FDA Approves AstraZeneca’s Imfinzi for Biliary Tract Cancer

Imfinzi, a checkpoint inhibitor developed by AstraZeneca, has been approved by the FDA as the first immunotherapy for biliary tract cancer (BTC), a rare and aggressive form of cancer with few treatment options. Imfinzi (durvalumab) has been approved by the Food and Drug Administration for the treatment of locally advanced or metastatic biliary tract cancer in adults in combination with gemcitabine/cisplatin chemotherapy, extending Imfinzi’s use beyond lung cancer for the first time.

The approval follows the TOPAZ-1 trial, which demonstrated that adding Imfinzi to standard first-line chemotherapy for BTC increased overall survival compared to chemo alone without significantly increasing the side-effect burden. In the trial, roughly 25% of patients who received Imfinzi/chemo were still alive two years later, compared to 10% of patients in the chemo-plus placebo group.

Biliary tract cancer affects the bile ducts and gallbladder, with approximately 23,000 cases diagnosed annually in the United States. Life expectancy is low, with only 5% to 15% of patients expected to live past the age of 5. Because the disease can be asymptomatic in the early stages, patients frequently progress to the more severe disease before receiving treatment, when options are limited, and the prognosis is poor.

According to TOPAZ-1 lead investigator Aiwu Ruth He of Medstar Georgetown University Hospital in Washington DC, the improvement in survival seen with Imfinzi is significant, as this has not been seen for a new BTC therapy in many years. Imfinzi’s FDA-approved indications in NSCLC and SCLC helped drive sales of the PD-L1 inhibitor to USD 1.3 billion in the first half of this year. It has also been filed for BTC in the United Kingdom, Australia, Brazil, Canada, Israel, and Singapore, with an EU filing expected soon.

FDA Clears Boehringer Ingelheim’s Spesolimab for Generalized Pustular Psoriasis

Boehringer Ingelheim has received the first regulatory approval in the United States for spesolimab, an antibody therapy for the rare and life-threatening skin disorder generalized pustular psoriasis (GPP). The FDA has approved the IL-36 receptor inhibitor Spevigo to treat the devastating flares of painful blisters that are a feature of GPP. The flares can be severe enough to require patients to be hospitalized with complications such as heart failure, renal failure, and sepsis. The mortality rate after GPP flares ranges from 2% to 16%, with septic shock and cardiorespiratory failure being the leading causes of death. 

Spevigo is the first GPP therapy to be approved and the first IL-36 inhibitor to make it through development and onto the market. European regulators are also reviewing it, with a decision expected before the end of the year. The approval follows the recently published EFFISAYIL 1 trial, in which a single intravenous dose of spesolimab was compared to a placebo in 53 people with a GPP flare characterized by a high or very high density of pustules on the skin.

Around 54% of patients treated with spesolimab had no visible pustules on their skin a week after dosing, compared to 6% of those treated with placebo. According to the data, the protective effect lasted up to 12 weeks.

Other drugs that inhibit other cytokines, such as TNF, IL-17, and IL-23 inhibitors, are currently used off-label to help patients experiencing a flare, but there is limited evidence for their efficacy. The EFFISAYIL 1 trial was led by Mark Lebwohl of the Icahn School of Medicine at Mount Sinai in New York, United States, who described the approval as a “turning point” for patients with GPP and their clinicians.

Azurity Pharmaceuticals’ Konvomep Wins Another FDA Approval

Azurity Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) had approved Konvomep (omeprazole and sodium bicarbonate for oral suspension). Konvomep is approved for treating active benign gastric ulcers and reducing the risk of bleeding in upper gastrointestinal portion in critically ill patients.

“We are glad that patients will soon have access to this FDA-approved oral liquid formulation option of a commonly prescribed proton pump inhibitor,” said Richard Blackburn, CEO, Azurity Pharmaceuticals. “Patients are our priority, and our aim is to bring them new formulations that help them benefit from established medicines. Konvomep may offer patients, particularly patients having difficulty swallowing pills or capsules, an option for treatment tailored to their needs.”

“Patients who struggle with taking solid oral dosage forms may be overlooked and have historically had limited FDA-approved treatment options available as liquid formulations,” said Olga Hilas, Professor, Clinical Health Professions, St. John’s University College of Pharmacy & Health Sciences, Queens, New York.

It is expected that Konvomep will become commercially available in pharmacies nationwide in first quarter of 2023.

Amgen’s Lumakras Shows Promising Results in KRAS-mutated Non-small Cell Lung Cancer (NSCLC) in a Phase III Trial

Amgen announced that the global Phase III CodeBreaK 200 trial accessing once-daily oral LUMAKRAS (sotorasib) met its primary end-point of progression-free survival, illustrating statistical significance and superiority over the standard of care chemotherapy, intravenous docetaxel. The first randomized clinical trial for a KRASG12C inhibitor evaluated the efficiency and safety of LUMAKRAS in approximately 340 previously treated patients suffering with KRAS G12C-mutated non-small cell lung cancer who received at minimum prior platinum-based doublet chemotherapy and checkpoint-inhibitor therapy.

Amgen took on one of the difficult challenges of the last 40 years in cancer research by developing LUMAKRAS/LUMYKRAS, a KRASG12C inhibitor. LUMAKRAS/LUMYKRAS has demonstrated a favourable benefit-risk profile with rapid, profound, and durable anti-cancer activity in patients suffering with locally advanced or metastatic non-small cell lung cancer harboring the KRAS G12C mutation with a once-daily oral formulation.

Amgen is progressing the most extensive and broadest global KRASG12C inhibitor development program with unparalleled speed and exploring more than ten sotorasib combination regimens, including triplets, with clinical trial sites spanning 5 continents. Upto date, around 6,500 patients around the world have received LUMAKRAS/LUMYKRAS through the clinical development program, expanded access and commercial use. LUMAKRAS/LUMYKRAS is Being Studied in Various Other Solid Tumors

Lung cancer is the main cause of cancer-related deaths globally, and accounts for more deaths worldwide than breast cancer, prostate cancer, and colon cancer combined. Overall survival rates for non-small cell lung cancer are improving but remain poor for patients suffering with advanced disease, and 5-year survival rate is only 8 per cent for those with metastatic disease.

The CodeBreaK clinical development program for Amgen’s drug sotorasib is directed to study patients with an advanced solid tumour with the KRAS G12C mutation and address the longstanding unmet medical need for these cancers. 

Novo Nordisk to Acquire Forma Therapeutics for USD 1.1 Billion

In one of the major acquisition updates in the healthcare industry, Novo Nordisk has announced that it has signed an agreement to acquire Forma Therapeutic. As per the definitive agreement, Novo Nordisk will acquire Forma Therapeutics for $20 per share in cash, which represents a total equity value of $1.1 billion. 

Additionally, Novo Nordisk will also take control of the Forma Therapeutics lead development candidate, etavopivat. Etavopivat is an investigational, once-daily, selective pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy with the potential to improve red blood cell health and transform sickle cell disease treatment scenarios. Novo Nordisk, over the past 40 years, has been actively working to develop and deliver transformative medicines to patients around the world with rare and devastating diseases. The accession of Etavopivat will greatly complement and accelerate Novo Nordisk’s strategy to grow its scientific presence and pipeline in hemoglobinopathies, a group of disorders in which there is abnormal production or structure of the hemoglobin protein in the red blood cells.

Forma Therapeutics is a clinical-stage biopharmaceutical company actively engaged in the research, development and commercialization of novel therapeutics for the treatment of rare hematologic diseases and cancers, including sickle cell disease (SCD). Forma Therapeutics has outstanding clinical development capabilities and generated a broad proprietary portfolio in the hematologic drug development segment based on differentiated mechanisms of action. The acquisition of Forma Therapeutics is expected to enhance Novo Nordisk’s market position in the sickle cell disease therapeutics market.

Sanofi’s Xenpozyme Becomes First FDA-Approved Drug ASMD

In the drug regulatory updates segment, the US FDA has approved Sanofi’s XenpozymeTM (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), often referred to as Niemann-Pick disease, in adult and pediatric patients. With the FDA’s approval, Xenpozyme is the first therapy approved specifically for treating ASMD and is currently the only approved treatment for this disease.

The approval of Xenpozyme is based on the positive outcome from the ASCEND and ASCEND-Peds clinical trials. In the ASCEND and ASCEND-Peds clinical trials, Xenpozyme demonstrated clinically suitable improvement in lung function, platelet count, and reduction of spleen and liver volumes, with a demonstrated safety profile. Xenpozyme is approved only for ASMD symptoms unrelated to the central nervous system.

ASMD is an extremely rare, progressive genetic disease that results from a deficiency of the enzyme acid sphingomyelinase and it leads to a significant morbidity and mortality. As per the estimates, fewer than 120 patients are diagnosed with ASMD in the US, and nearly two-thirds of patients with ASMD are children. Moreover, some of them survive only until two or three years of age. As of now, the management of ASMD includes supportive care to address the impact of individual symptoms. However, the launch of the Xenpozyme is expected to improve the ASMD treatment scenario in the coming years immensely.

FDA Sets New Priority Review Date of Sanofi and Sobi’s Hemophilia A Drug Efanesoctocog Alfa 

US FDA recently gave Sanofi and Sobi a green flag for the approval of the hemophilia A drug candidate efanesoctocog alfa. The approval is due in the initial of 2023. Efanesoctocog alfa is a long-lasting hemophilia A that promises to deliver close to normal Factor VIII activity levels, potentially offering a promising relief for haemophilia A patients. Such tremendous therapeutic candidates demonstrate the advances in the medical segment and offer reliable cures, which may later transform the polylactic treatment landscape. 

Sobi and Sanofi, which occupy a considerable market size, are now planning to deliver an application seeking the approval of the hemophilia A drug candidate efanesoctocog alfa in Europe in 2023. The application will soon be applied once the outcomes from the phase III XTEND-Kids study (NCT04759131) can be seen. The drug was evaluated in 159 patients, mainly adults and adolescents 12 years or older. Phase III focuses on trials on young children with severe hemophilia A which occupy most of the diseased population. Previously efanesoctocog alfa was granted the Orphan drug designation by Europe, and the FDA earned efanesoctocog alfa the designation of breakthrough therapy, orphan drug and fast track. 

During the study, the results suggested that efanesoctocog alfa gradually reduced annual bleeding rates upto 77% compared to the preexisting therapies. Additionally, the drug entity showed reduced pain and improved joints. 

Neurocrine Bio Agrees USD 57 Million Takeover of UK Biotech Diurnal

Neurocrine Biosciences, known for potential capturing key players, has now decided to acquire UK Biotech Diurnal. The deal will ultimately increase the company’s potential for making therapeutic candidates for hormonal disorders. The deal was closed at a whopping 57 million. Diurnal, founded in 2004, is a key pharma player that majorly focuses on therapies for rare and chronic endocrine conditions. The potential drug candidates include those to cure hypothyroidism, hypogonadism, and other endocrine complexities. 

The deal will probably be finalised in the last week of October or the initial week of November. Neurocrine Biosciences is a major key player that occupies a considerable market size in tardive dyskinesia, Parkinson’s disease, endometriosis, and uterine fibroids segment. 

The pence per share of Duinal is also seen to boost upto 134%, accounting for 26.35 pence per share. The rise was seen soon after the company announced its with Neurocrine Biosciences. The shareholders of Durinal are expected to receive 27.5 pence per share as per the closing price on August 26. However, the deal is subject to terms and conditions per Durinal shareholders.  The ultimate price per share was doubled after the deal closed.