The Business Cocktail

Novartis takes on Regeneron with AMD drug that needs fewer doses than Eylea

Last year, U.S. sales of Regeneron’s drug to treat age-related macular degeneration (AMD) came in at $3.3 billion—68% of the company’s total revenues—so it’s no wonder investors have been expressing some concerns about potential rivals moving through the pipeline. Among those hoping to cut into Eylea’s market is Novartis, which is in phase 3 testing with its AMD candidate, RTH258. And if the latest data from those trials is any indication, Regeneron investors have good reason to worry. Novartis announced that in two head-to-head trials, RTH258 was as effective as Eylea, but in more than half of patients it could be injected in the eye every three months, versus monthly or every two months for Regeneron’s drug. The rate of side effects was comparable for the two drugs. Some analysts predicted Novartis would gain a competitive edge over Regeneron and the other major player in the field, Roche’s Lucentis.

Hospira recall of vials adds to woes of sodium bicarbonate shortage

Citing sterility concerns, Hospira, a subsidiary of pharma giant Pfizer, is voluntarily recalling vials used to inject sodium bicarbonate during surgery and in critical events. The nationwide recall adds to the ongoing shortage of the drug. The voluntary recall was announced on the FDA’s website and covers sodium bicarbonate and succinylcholine chloride vials that were found to have microbial growth. Such contamination can lead to sepsis. As part of the action, 42 lots of sodium bicarbonate vials were recalled along with five lots of succinylcholine chloride. Hospira said it is unaware of any reports of adverse events associated with the drug. In a separate statement, the regulatory agency said it is aware of the ongoing shortage situation affecting several injectable drugs manufactured by Hospira. Those include sodium bicarbonate injection (vials and syringes), dextrose 50% injection (vials and syringes), as well as emergency syringes of other drugs, including epinephrine, calcium chloride and atropine sulfate.

Takeda wraps up construction of new $111M solid dosage plant near Berlin

Japanese pharmaceutical giant Takeda said it has completed construction of its new $111 million manufacturing plant on the outskirts of Berlin in Oranienburg, Germany. Construction of the facility was announced by Takeda in 2014 as part of the company’s plan to streamline the global manufacturing of its solid dosage form pharmaceutical products from its plant in Osaka to the Oranienburg facility and Hikari plant in Japan, the company said. The 21,400-square-meter Oranienburg plant is scheduled to open by the end of the year and has already created 180 new jobs. Production at the new facility is expected to supply about 100 countries globally and will include drugs used to treat diseases for in the areas of gastroenterology, central nervous and cardiovascular systems. Germany has been the focus of a lot of Takeda manufacturing investment. In November, the drugmaker said it would “immediately” begin construction on a $106 million plant at its manufacturing site in Singen, Germany. That plant, which is expected to be completed in 2019, will focus on Takeda’s big push into a vaccine to treat dengue.

FDA bans imports of Ipca Lab drugs

The FDA has banned the import into the U.S. of drugs manufactured at three facilities in India operated by Ipca Laboratories as a result of ongoing concerns about data manipulation and falsification of records. The facilities include Ipca’s API plant in Ratlam, its formulations plants in Pithampur in Madhya Pradesh and Piparia in Silvassa, which have been under U.S. regulatory scrutiny for several years. The company halted product shipments from the trio of plants after the FDA put them on an import alert list in 2015 following inspections conducted in 2014. In the stock exchange filing, Ipca said it received notification from the FDA that drugs manufactured at three facilities won’t be allowed into the U.S. until the company can demonstrate that products from the sites are in compliance with prescribed norms. The news sent Ipca’s stock down 15% last Friday.

The Business Cocktail

AstraZeneca offloads headache drug Zomig to Grünenthal to raise $300M cash

AstraZeneca had some positive news to announce at ASCO about its PARP Lynparza for treating breast cancer, but all in all, the U.K. company continues to struggle and so will sell yet another asset to raise cash while CEO Pascal Soriot works to right the ship. The U.K drugmaker today said that it has a deal to sell Germany’s Grünenthal rights to its migraine treatment Zomig for all markets outside of Japan. AZ will get $200 million when the deal is done and up to $102 million in additional payments if targets are hit. The arrangement includes rights to the drug in the U.S. where it has been licensed to Impax Pharmaceuticals since 2012. Impax will continue to market Zomig in the U.S. AstraZeneca will continue to manufacture and supply it, during the transition.  Grünenthal’s experience with pain drugs puts them in a good position to serve patients that rely on the headache med, Mark Mallon, executive VP of global product & portfolio Strategy at AstraZeneca said in a statement.

Pfizer’s $14B Medivation deal’s now a cautionary M&A tale, thanks to ASCO

The annual ASCO meeting often throws new light on old drugs—and old dealmaking. This year, it’s last summer’s most ballyhooed M&A deal—Pfizer’s $14 billion buyout of Medivation—that doesn’t look so impressive anymore, thanks to data rolled out over the weekend. Big pharma and big biotech bidders swarmed on Medivation back then, partly because of its pipeline med talazoparib, a PARP inhibitor then-Medivation chief David Hung touted as a best-in-class med. The other big draw was Xtandi, the blockbuster-to-be prostate cancer drug, and its present-day cash flow. The two together added up to offers from Pfizer and Sanofi, plus a range of other bidders rumored to include Amgen and Gilead Sciences. Thing is, Xtandi competes head-to-head with Johnson & Johnson’s Zytiga, and while Xtandi put up disappointing data in a study that might have helped grow sales, Zytiga racked up a couple of study wins at ASCO that analysts now say could help the J&J med zoom ahead on the indication front.

Takeda starts work on €40M plant to produce multiple myeloma drug Ninlaro

Japanese drugmaker Takeda has started work on a new plant to produce its oral multiple myeloma drug Ninlaro, a first-to-market active proteasome inhibitor, and expects the facility will be ready to ship the potential blockbuster next year or in early 2019.  The company says construction begins this month on the €40 million ($42.8 million) facility at its Grange Castle site in Dublin to manufacture Ninlaro. The plant is slated to be completed in the second quarter of 2018 and be operational to ship secondary packaged product in the second half of fiscal 2018, which ends on March 31, 2018. Ireland development authorities boasted in December that Takeda was expanding in the country, but the drugmaker is now offering up more details on the project. It said the 5,672-square-meter (61,053-square-foot) production facility will house the API, formulation, primary and secondary packaging and quality-control processes. The company will add 40 jobs at the site as a result of the expansion.

Germany’s doc payment transparency effort relies on honesty

In the U.S., the Open Payments database lays out pharma’s payments to doctors for all to see. In Germany, where disclosure isn’t mandated as it is in the U.S., a nonprofit is trying transparency based on the honor system. German doctors will be able to voluntarily disclose contributions from pharma in a database compiled by the nonprofit journalism organization Correctiv, according to Germany’s DW. About 71,000 doctors in the country received €575 million in cash or in-kind contributions from the drug industry last year, a Correctiv investigation found in December. That’s far less than the $7.52 billion that drug and device makers funneled to doctors and healthcare providers in the U.S. in 2015, the most recent data available, but slightly more than the £345 million that changed hands in the U.K. Making those payments public at the doctor-by-doctor level is important, advocates say, because studies show financial relationships between drug makers and doctors can affect physicians’ prescribing habits.

 

The Business Cocktail

Tesaro and its new med Zejula are on the block, but bidders aren’t rushing in

Just ahead of the year’s biggest download of cancer data, the maker of a closely watched drug has put itself up for sale, according to The Wall Street Journal. That’s Tesaro, whose PARP inhibitor Zejula won FDA approval in March. That med launched soon after at a price of $118,000 per year, which Tesaro touted as lower than its head-to-head rivals in ovarian cancer, AstraZeneca’s Lynparza and Clovis Oncology’s Rubraca. Analysts have pegged Zejula, or niraparib, as one of 2017’s biggest launches, with 2022 sales of $1.9 billion. That’s an enticing prospect for bigger companies looking to beef up their oncology businesses, but that very fact could make it an expensive prospect, too. Thanks to a big stock runup since Tesaro rolled out top-line Zejula data last July, the company’s market value as of Thursday morning was $8 billion. Tesaro has requested offers from a variety of potential bidders over the past few weeks—but they also say potential acquirers aren’t exactly falling over themselves to pursue a deal. Price is potentially one reason, obviously. Another? Would-be buyers might be waiting for the American Society of Clinical Oncology meeting this weekend, where AstraZeneca will present data on Lynparza and Tesaro will post data on a Zejula combination with Merck’s blockbuster PD-1 therapy Keytruda. Still another reason for the supposedly lukewarm interest: Pfizer’s $14 billion deal for Medivation—and its PARP med talazoparib—has come in for criticism lately.

Mylan lowballed Medicaid on EpiPen by $1.27B

Mylan quickly agreed to a $465 million settlement with the U.S. government last fall in a move to put some of its EpiPen problems in the past. Now, it appears that figure is far short of taxpayer harm resulting from the injector’s misclassification on Medicaid, news that comes as the deal has yet to be finalized. The Department of Health and Human Services’ Office of Inspector General reports that EpiPen’s Medicaid misclassification cost taxpayers $1.27 billion from 2006 to 2016, far exceeding the U.S. government’s settlement agreement with the drugmaker. Under heavy scrutiny for years of price hikes on the lifesaving epinephrine injection, Mylan struck a deal with the government to resolve fresh allegations of Medicaid misclassifications back in October. Pushing to learn more about the misclassification, Sen. Grassley on Wednesday said Mylan still isn’t cooperating with the investigation. Mylan is withholding communications between it and the Centers for Medicare and Medicaid Services, which notified the company several times about the misclassification, according to the senator.

Ohio sues drugmakers for marketing fraud

After seeing drug overdoses become the state’s leading cause of accidental deaths, officials in Ohio have had enough. In a new lawsuit filed on Wednesday, Ohio Attorney General Mike DeWine is going after Teva, Allergan, Johnson & Johnson, Purdue and Endo for alleged “fraudulent marketing practices” on powerful opioid painkillers. Ohio’s 101-page lawsuit says the pharma companies, individually and together, broke pharma marketing rules and “helped unleash” an opioid epidemic that has had “far-reaching financial, social, and deadly consequences” in the state. The companies did that, according to the suit, by spending millions to widely market the meds while downplaying risks. Similar to a host of other lawsuits filed against opioid makers, Ohio’s suit says that to push their painkillers, the companies borrowed a page from the “Big Tobacco playbook.” They worked to convince “key opinion leaders” and professional societies of the benefits of opioids in treating chronic pain. For its part, Teva said it’s reviewing the complaint and can’t comment until that review is complete. Endo said it couldn’t comment on ongoing litigation; Allergan also declined to comment.

ATUM, Horizon Discovery Announce cross-license agreement

ATUM and Horizon Discovery have announced that they have signed a cross-license agreement for Horizon’s CHO SOURCE platform and ATUM’s vector technology to speed development of highly productive stable cell lines for drug development. ATUM has licensed Horizon’s CHO SOURCE platform, including the Glutamine Synthetase (GS) Knock-Out CHO K1 (Chinese hamster ovary) line, and will use its proprietary Leap-In® Transposase Technology to offer cell line development services.  Horizon has exclusively licensed a vector suite developed by ATUM for the CHO SOURCE platform, to provide a complete cell line solution to its customers. Together, these technologies enable expression of complex biologics for customers of both ATUM and Horizon. ATUM is also offering Horizon customers a no-fee evaluation license for the transposase system.

 

The Business Cocktail

Shadowed by nearly $30B in debt, Valeant’s $50M guidance raise looks pretty paltry

Faced with a crippling pile of debt, Valeant on Tuesday slightly increased its 2017 earnings guidance, one small positive development as it works to rebound from its noted downfall. The Canadian drugmaker reported first-quarter revenues of $2.11 billion on Tuesday, missing FactSet consensus estimates of $2.19. But the company sees things going well enough to increase its 2017 earnings guidance to between $3.6 and $3.75 billion, slightly increased from a previous range of $3.55 billion to $3.7 billion. The move triggered a 20% run-up in share price by mid-morning Tuesday, but at least one analyst urged caution. Wells Fargo’s David Maris pointed out that “that the $50 million raise is relative to approximately $28.88 billion of total debt.”

Sanofi vows to limit 2017 price hikes to 5.4% as part of new policy

Sanofi may be late to the pricing-pledge party, but it’s come up with some benchmarks that could have other drugmakers defending their own. Rather than limiting price hikes to less than 10%, the French drugmaker promises to keep any price increases at or below an official health inflation measure that’s projected to hit 5.4% in 2017. The company will also report numbers on its aggregate price increases annually, disclosing hikes to gross prices and net prices, which reflect rebates and discounts granted to payers. Sanofi’s policy follows more than a year of intense scrutiny on pharma pricing in the U.S. as high-profile hikes turned a spotlight on the entire industry. Prompted by leaps in the cost of Mylan’s EpiPen, a set of Valeant Pharmaceuticals’ bought-in drugs, and, notoriously, a massive price hike on Daraprim engineered by then-Turing Pharmaceuticals CEO Martin Shkreli.

Top pharmas team with Express Scripts to gin up savings for cash-paying patients

As pharmacy benefit managers and drug makers continue to clash over their respective roles in controversial drug-price hikes, eight top pharmas and leading PBM Express Scripts have teamed up to offer cheaper meds to patients forced to pay cash for their prescriptions. Through a collaboration that comprises drug companies, Express Scripts, 40,000 pharmacies and tech partner GoodRx, the program is designed to make drugs affordable for patients with high-deductible insurance plans, or no insurance at all. The effort could help quell public outcry over drug pricing by limiting the hit to patients’ pocketbooks. For instance, Mylan’s EpiPen pricing scandal hit after parents buying back-to-school EpiPens faced big increases in their out-of-pocket costs—and those parents took to social media to air their grievances. So far, the Inside Rx program has attracted a who’s who among drugmakers, mostly weighted toward those headquartered outside the U.S.

PhRMA expels 22 members with new R&D rules as it works to burnish its image

Industry lobbying group PhRMA has cleared its decks of nearly two dozen members as it tries to distance itself from those companies most likely to catch heat over drug pricing practices. While not all of those who were dropped have price-pushing reputations, the practices of some are particularly notorious for their methods. The organization did this with new bylaws that mandate members meet minimum R&D investment requirements, a move that was expected to force out smaller companies whose business model is built more on buying drugs and then jacking up prices significantly. The move comes as the lobbying group works to portray its membership as serious drugmakers that are investing heavily in innovative new medications.

Notizia

Lilly moves forward with biologics plant expansion in Ireland

Eli Lilly has confirmed that it will move forward with plans to add a manufacturing line at its site in Kindsdale, County Cork in Ireland. The construction might begin in coming months and commissioning will begin in 2019. The expansion is expected to add about 130 workers to the site by 2020. As negotiations are underway, specifics of the investment are not being released. Earlier reports put the cost at about €200 million. The site also does API production for a host of small molecule drugs. Last year, the company said it would invest nearly $40 million (€35 million) to build a continuous manufacturing facility. Tremors of concern spread through Ireland in February when Lilly said it halted preparations for the project. That ignited speculation among some politicians that Lilly might divert funds to the U.S. after President Trump held a meeting with pharma execs in which he urged them to invest in U.S. manufacturing.

Brazilian authorities raid Alexion office in another sales-practices probe

Alexion’s sales practices are under the lens again. Just four months after concluding its top brass inappropriately pressured staff to pad Soliris sales in the U.S., Brazilian authorities raided the company’s Sao Paulo offices as part of an investigation into its marketing there. Brazil’s police are probing whether the Connecticut-based biotech, helped by Brazilian patient association Associacao dos Familiares, Amigos e Portadores de Doenças Graves (AFAG), subsidized lawsuits for patients to gain access to an Alexion drug through Brazil’s national health system. AFAG, to which Alexion has said it gives financial support, was also searched. Law enforcement officials have turned up evidence showing that some of the lawsuits—which number more than 900 in the last six years—were fraudulent and used fake diagnoses to help reel in patients. Through those lawsuits, Alexion drummed up $400 million in sales from the Brazilian government in that time period, the warrant request says.

Bristol-Myers, AstraZeneca in legal hot seat over Onglyza heart failure warning

Last year, the FDA called for stronger heart-failure warnings on the label of AstraZeneca Type 2 diabetes med Onglyza. And now, AstraZeneca and former partner Bristol-Myers Squibb are being hit with 14 lawsuits related to the med’s heart failure risks. The suits, filed in the District of New Jersey, claim the drug makers failed to warn users that Onglyza and related combo product Kombiglyza XR can cause cardiac arrest, congestive heart failure, and death. The pharma pair began marketing Onglyza in 2009 before conducting clinical trials to see whether it upped patients’ cardiac risks, the plaintiffs allege, ignoring 2008 FDA guidance that urged companies to “demonstrate that” new therapies “will not result in an unacceptable increase in cardiovascular risk.” AstraZeneca, for its part, “is confident in the safety and efficacy of Onglyza, when used in accordance with the FDA-approved label, which has been established through clinical trials. We will vigorously defend against the allegations made by the plaintiffs,” a spokeswoman said in a statement.

PhRMA expected to weed out some members with new R&D rules

PhRMA, which has already rid itself of a couple of members who had brought unwanted attention to the industry for their practice of jacking up prices, will vote on new membership rules that are expected to trim its membership, and hopefully criticism, even further. PhRMA spokeswoman Holly Campbell said today she could not discuss details of the new rules ahead of tomorrow’s vote. The group began reviewing its bylaws to focus on “research-based biopharmaceutical companies who take significant risks to bring new treatments and cures to patients,” as public criticism over drug pricing has escalated. PhRMA’s new rules will require members to invest at least $200 million a year on average over three years of research and development. Their R&D budgets would also have to equal at least 10% of their global sales.

Notizia

FDA Approves Alunbrig (brigatinib) for Rare Lung Cancer

Takeda Pharmaceuticals announced the FDA approved Alunbrig (brigatinib) to treat patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib. Brigatinib is a kinase inhibitor that can be taken orally. The recommended dose is 90 mg orally once daily for the first 7 days. If 90 mg is tolerated during the first 7 days, the patient should increase the dose to 180 mg orally once daily. The pill can be taken with or without food. Brigatinib has an orphan drug designation for this indication and Takeda received an Accelerated Approval of the drug based on tumor response rate and duration of response. An Accelerated Approval means Takeda can market the drug now but additional studies (i.e., a phase 3 clinical trial) may be needed by the FDA moving forward.

AZ nabs FDA nod for bladder cancer for checkpoint med Imfinzi

AstraZeneca got its first FDA approval with its checkpoint inhibitor Imfinzi (durvalumab), as the British pharma giant picked up an accelerated green light in urothelial carcinoma. This makes the company the third Big Pharma player to enter the advanced bladder cancer space with a PD-1/PD-L1 inhibitor. Imfinzi will take on established player Tecentriq from Roche, which hit the scene last May, and Opdivo, the Bristol-Myers Squibb entrant that won its bladder go-ahead in February. Merck is still chasing a bladder cancer nod for its contender, Keytruda. Imfinzi, though, will roll out at a higher price point than its nemeses; as estimated average monthly cost of the med will be $15,000, compared with its rivals’ $12,500. The approval comes based on a single-arm, 182-patient trial, which produced an overall response rate of 26.3% among the 95 patients with high PD-L1 scores. As Tecentriq does, Imfinzi goes hand in hand with a complementary diagnostic from Ventana that assesses PD-L1 levels in tumors.

Novo Nordisk to settle med-whistleblower suit

Novo Nordisk has moved to settle a whistleblower lawsuit alleging the company ran a “white-coat marketing scheme” to pump up sales of NovoLog, Victoza and Levemir. According to the suit, recently unsealed as part of the settlement process—Novo Nordisk partnered with a clinical education company, Healthstar’s PT, to set up a program dubbed Changing Life with Diabetes. The group hired and trained certified diabetes educators (CDEs), according to the suit, which was brought by two former managers and more than two dozen states. The whistleblowers claim that since 2006, Novo ran the program to gain crucial access to physician practices, where CDEs would provide thousands of dollars’ worth of educational programs and materials. The idea behind the program, according to the suit, was to induce prescribers to write scripts for Novo’s diabetes meds by offering such services. Due to this, U.S. pricing pressures have taken a toll at Novo lately, forcing the company to cast off 1,000 staffers last fall.

Novartis to pay $50M, lose some drug coverage in Korean bribery probe

For more than a year, Novartis has been under investigation in Korea for allegedly bribing doctors to pump up sales. Now, the Swiss drug giant is learning about its punishment in the country as authorities there decided to issue a fine of nearly $50 million. On a preliminary basis, Korea’s Ministry of Health & Welfare (MHW) has fined Novartis 55 billion Korean won, approximately $50 million, and suspended reimbursement of Exelon and Zometa for three months. The final decision is expected by the end of May. Previously, authorities were weighing a suspension of the company’s big-selling cancer med Glivec, according to The Korea Times, but feared that move would leave some 3,000 to 5,000 chronic myeloid leukemia patients with no other options.

Notizia

Samsung biosim nod sets J&J’s Remicade up for tough fight

Samsung Bioepis won FDA approval late Friday for its Remicade substitute, Renflexis (infliximab-abda), becoming the the second biosimilar to threaten that brand in the U.S. Not an unexpected event, and the launch isn’t expected until October because of biosimilar rollout rules. But six months pass quickly in the pharma world, and analysts see the second biosim emboldening payers to press for bigger rebates and pushing the three contenders to get more aggressive about winning share—or protecting it, as the case may be. Add into the mix the fact that Merck & Co. has marketing rights and the rivalry looks even more interesting. Soon, Remicade will have companies experienced on both sides of the biosimilar-brand competition making a run at its U.S. sales.

Lilly’s cancer med abemaciclib hits survival mark, but can it stand up to Ibrance and Kisqali?

Eli Lilly’s chances of snagging an abemaciclib approval as wide as those of its rivals are looking even better. In a phase 3 trial of the drug in previously untreated patients with HR-positive, HER2-negative breast cancer, abemaciclib nailed its primary endpoint, demonstrating significant improvement in progression-free survival. The performance follows up on last month’s positive results from Lilly’s other key trial, a test in patients whose cancer had come back after a first round of treatment. With those two sets of positive data in hand, the Indianapolis drugmaker is “assured of a product label with the same breadth” as that of Pfizer’s Ibrance and Novartis’ Kisqali, Bernstein analyst Tim Anderson predicted in a Monday note to clients. But that may not be enough to ensure abemaciclib’s success.

BioMarin sees ‘high end’ pricing on Brineura ahead of FDA decision

Children with a rare, devastating brain disease may soon have their first treatment option in BioMarin’s Brineura, up for an FDA decision later this week. And it’s likely to instantly join the ranks of the world’s most expensive meds if approved. Experts from the European Medicines Agency’s Committee for Medicinal Products for Human Use on Friday endorsed the drug to treat Neuronal Ceroid Lipofuscinosis Type 2 disease, a form of Batten disease. The EMA usually green-lights drugs that have CHMP recommendations, but it isn’t required to do so. In the U.S., Brineura is set for a decision date the coming Friday. Execs at BioMarin said the company is in label discussions with the U.S. agency.

Biogen’s Spinraza scores later-onset SMA data for stepped-up push with payers

Hoping to win over careful payers, Biogen has new data to support its superpricey spinal muscular atrophy (SMA) drug Spinraza. In new phase 3 data from a study in children with later-onset SMA, patients on Spinraza “demonstrated a highly statistically significant and clinically meaningful improvement in motor function” over their counterparts who received a sham treatment, the Cambridge, Massachusetts-based biotech said in a Monday release. Biogen won approval for the med back in December and sparked criticism by pricing the groundbreaking injection at $750,000 for the first year, a figure that falls to $375,000 for subsequent years. In an earlier study of infantile-onset (type 1) patients, a higher percentage of children on Spinraza survived compared with untreated patients. Data from the newly finished study, Cherish, weren’t available at the time, but the FDA nonetheless approved the med for treatment of all SMA types.

Klick Labs creates empathy-boosting Parkinson’s simulator

Klick’s digital innovation unit Klick Labs created a device to transmit tremors from a Parkinson’s patient to someone who doesn’t suffer from the disease, to dramatically illustrate its effects. That’s empathy-creation in action, and the basis for a planned clinical study about the ways empathy can improve patients’ health. For pharmas, it’s a potential tool for sales-rep training. And the device can show differences between patients who are treated and those who aren’t, Klick says, opening up the potential for other applications. The programmable SymPulse device connects a Parkinson’s patient to a second person. A sensor on the patient digitizes and transmits tremors or muscle contractions, using electrical muscle stimulation, to a device worn by the non-patient. The result? The receiver can feel the exact same things the patient does. Klick refers to it as “tele-empathy.”

Notizia

Malaysia calls for phase 4 Dengvaxia study before considering full approval

Already struggling to meet initial expectations, Sanofi Pasteur’s dengue vaccine Dengvaxia will have to undergo phase 4 testing in Malaysia before the endemic country agrees to sign off on a full approval. Malaysia’s National Pharmaceutical Regulatory Agency’s Drug Control Authority, which conditionally approved the vaccine, said in a notice that if the study fails to verify the clinical benefit, DCA may withdraw Dengvaxia’s registration. The French vaccine maker will conduct the two-year trial jointly with the country’s Ministry of Health, aiming to further assess the vaccine’s effectiveness and safety. Before the readout, the vaccine will only be available to eligible trial volunteers 9 to 45 years old. It won’t be covered by Malaysia’s National Immunisation Programme, meaning that participants will need to pay to get vaccinated.

Bristol-Myers’ Opdivo racks up latest NICE rejection, this time in head and neck cancer

Bristol-Myers Squibb’s Opdivo hasn’t even won marketing approval for head and neck cancer in England and Wales yet, but their cost watchdog is already shutting it out. The National Institute for Health and Care Excellence on Tuesday introduced the latest in a series of setbacks for the BMS immuno-oncology hotshot: draft guidance that doesn’t recommend it as a head and neck cancer therapy. The med’s costs—£439 per 40-mg vial and £1,097 per 100-mg vial, less a confidential discount agreed to with Bristol-Myers—“were considered to be very high in relation to its benefit to be recommended for routine NHS use at present,” Carole Longson, director of NICE’s health technology evaluation center.

FDA issues another Mylan plant in India a warning letter

Less than two years after being given a warning letter by the FDA for problems at three plants it got in its buyout of a sterile injectables specialist Agila Specialties, another Mylan plant in India has been issued a warning letter for ongoing data integrity issues. In the warning letter, issued last week and posted by the FDA today, the agency said investigators continued to find issues with batch testing results disappearing from computers when failed tests were involved. The observations were based on an inspection of the finished pharmaceutical plant in Maharashtra, India. It noted that even though the facility invalidated 101 of 139 initial out-of-specification (OOS) assay results, about 72%, employees never thoroughly investigated to find the root cause of the issues and didn’t include them in the results reported to the agency.

Mexican regulator undermines Teva’s Rimsa fraud claims with ‘all clear’ memorandum

Teva contends that Mexican generics buy Rimsa sold defective, illegal products and lied about it, duping not only itself as buyer, but regulators and the public as well. But a new memorandum from Mexico’s drug watchdog doesn’t help its case. According to a March 13 document seen by The Times of Israel, Mexico’s Federal Commission for the Protection against Sanitary Risk (COFEPRIS) found no “unexpected adverse effects” among Rimsa’s 147 products and has located authorization for all of them within its archives. Sixteen COFEPRIS checks on Rimsa dating back to 2009 also failed to turn up any issues that could pose health risks, and the body found documentation provided by Rimsa to be “truthful and correct.” The conclusions don’t exactly support Teva’s legal claims that Rimsa withheld discrepancies between manufacturing processes and descriptions in product registrations filed with regulators.

Mission bags Fox Foundation grant for Parkinson’s program

Mission Therapeutics and the University of Oxford have landed a grant to fund testing of USP30 inhibitors. The Michael J. Fox Foundation for Parkinson’s Research (MJFF) is putting up the money to enable Mission to test its USP30 inhibitors in stem cell-derived Parkinson’s disease models. MJFF is funding the research in an attempt to validate USP30 as a target for the treatment of Parkinson’s. By inhibiting USP30 in malfunctioning mitochondria, the scientists stand to show whether the therapeutic approach may improve the operation of the organelles and neuron health. And there is an expectation this will enable the identification of biomarkers of USP30 that will prove useful in subsequent clinical development.

Hemophilia A- Market Scenario

Hemophilia is the most common inherited bleeding disorder which leads to spontaneous bleeding as the blood does not clot properly. Hemophilia, characterized by the spontaneous bleeding and swollen joints due to bleeding into the joints, is of several different types- such as Hemophilia A, Hemophilia B, and Hemophilia C. Hemophilia A is the most common type, which occurs due to the deficiency or decrease of factor VIII- a factor that plays a major role in clotting blood. The severity of Hemophilia A depends on the presence of plasma levels of factor VIII.

According to the National Heart, Lung, and Blood Institute (NHLBI), Hemophilia A is observed among 8 out of 10 patients with Hemophilia. Hemophilia A (factor VIII deficiency) is four times as common as Hemophilia B (Factor IX deficiency).

 

There are around 20 drugs which are being marketed worldwide for the treatment, prophylaxis or management for Hemophilia A. Though Kogenate FS (developed by Bayer HealthCare) lost its patent, still, it is the top-selling drug of Hemophilia A contributing to the majority of the market size. The market is also dominated with several recombinant proteins developed by the major players such as Baxter, Bayer, and Pfizer. Among major players, Shire Plc is the most progressive company which has recently been acquired Baxalta in June 2016.

Advate is also leading the race for Hemophilia A.  Revenues of Advate have had a major impact on the market. Development of novel coagulating factors, technological advancement, and advancement in diagnosis techniques are stimulating the growth of the market.


Additionally, Baxalta (now Shire) is also facing tough competition from the Biogen IDEC with the launch of long-acting therapies such as Eloctate for Hemophilia A.  Bayer has recently received approval Recombinant Factor VIII, Kovaltry in 2016 which is expected to fuel the market growth for the forecasted period. Hemophilia A market is expected to increase due to upcoming Hemophilia therapies which shall be launched in 2019 (Turoctocog alfa pegol for Hemophilia A and LR769 for Hemophilia A and Hemophilia B) and 2021 (Emicizumab and Damoctocog alfa pegol for Hemophilia A).

Insight by:
Sukhvinder Singh
Associate Analyst

DelveInsight is a leading Business Consulting and Market Research Firm. We help our clients to find answers relevant to their business, facilitating their decision-making. DelveInsight also serves as a knowledge partner for business strategy and market research. We provide comprehensive analytical reports across various therapeutic indications. DelveInsight has a database of 3000+ high-quality analytical reports.

The Business Cocktail

Valeant’s asset-sale woes continue as Stada, Mundipharma lowball iNova bids

Embattled Valeant has promised its investors $8 billion in asset sales—but as the company’s latest deal struggles continue to demonstrate, getting there may not be so easy. The bids rolling in for Valeant’s Australian iNova subsidiary aren’t quite up to expectations, with a private equity consortium comprising The Carlyle Group and Pacific Equity Partners, as well as fellow drug makers Mundipharma and Stada, each launching offers of around $900 million. Now, the Review says, Valeant is weighing whether to hang onto iNova—which sells products ranging from weight-loss drug Duromine to asthma treatment Qvar—after the Goldman Sachs-run sales process. Late last year, Valeant also reportedly tried to sell its underperforming GI unit, Salix, to Japan’s Takeda, but talks on a $10 billion deal ultimately fell through over price.

Sanofi Pasteur coughs up $19.8M to settle claims it overbilled the VA

Sanofi Pasteur agreed to pay more than $19.8 million to settle allegations that it overcharged the Department of Veterans Affairs for its products. The French pharma’s vaccines unit voluntarily reported the “calculation and reporting error” with the VA in 2012, and has since “cooperated fully and negotiated in good faith with the government,” according to a statement from the company. Sanofi Pasteur based its first disclosure to the U.S. government on products sold to the VA from 2007 to 2011. A follow-up investigation by the VA’s Office of Inspector General found the overcharging error dated back to 2002, the DOJ said in a statement. Sanofi said that, for some products, the miscalculation led to a lower price to the VA. The company will not seek reimbursement for those undercharges, it said. Neither the DOJ nor Sanofi identified the products involved.

With key rollouts looming, Sanofi’s Genzyme chief Meeker hands over the helm

As Sanofi gears up its biggest launch of the year, the Genzyme chief tasked with overseeing that rollout is leaving the company. David Meeker, CEO of the U.S.-based Sanofi unit and EVP overall, will exit as of June, with Bill Sibold—Sanofi Genzyme’s head of multiple sclerosis, oncology and immunology—taking his place. Sibold’s ascendance comes as Sanofi Genzyme grows in importance for the France-based drug maker. Pegged as one of the company’s “growth platforms” from the days after ex-CEO Chris Viehbacher engineered the acquisition, Genzyme is shouldering an even bigger load now. It’s another in a series of executive changes at Sanofi as well, as CEO Olivier Brandicourt, who took the helm in February 2015, shuffles his team and the divisions they lead.

India’s Alkem says the FDA has approved upgrade plans for cited API plant

After a year of nothing but bad news about FDA citations of its plants, India’s Alkem Laboratories has announced something positive. The Indian drugmaker said that the FDA has issued an Establishment Inspection Report, signing off on Alkem’s plans to upgrade operations at its API plant in Ankaleshwar, India. The plant had been cited with a Form 483 with three observations during a visit by the FDA in December. The drugmaker has 14 manufacturing sites in India and the FDA has been doing a series of inspections after being alerted to problems by U.K. regulators who claimed the drugmaker had used fake data in clinical trials of an unspecified antibiotic and brain disorder drug. That alert came after the FDA had serious issues with Alkem shipping unapproved drugs to the U.S.

GSK recalling nearly 600,000 Ventolin inhalers in U.S.

With sales of its respiratory drugs doing well, GlaxoSmithKline has been spending hundreds of millions of dollars to expand manufacturing of several kinds of inhalers. But a glitch at one of its Ventolin inhaler plants has resulted in recalling nearly 600,000 units in the U.S. According to an FDA Enforcement Report, GSK recently began recalling 593,088 Ventolin inhalers after discovering that an elevated number of the units were out of specification for leak rate, the company reported. The units were manufactured at its plant in Zebulon, North Carolina. A GSK spokesperson pointed out this was not a consumer level recall, so patients can keep any Ventolin inhalers they have on hand. The voluntary recall is to the retail and wholesaler level so products are being removed from those channels.