Although COVID-19 continued to dominate headlines in 2021, pharmaceutical companies did not cease developing new treatments this year. The US Food and Drug Administration maintained a rapid rate of new drug approvals this year, all while managing the urgent examination of COVID-19 tests, treatments, and vaccines under development. The FDA authorized a number of novel drugs that address previously unmet medical needs or dramatically improve patient quality of life. In 2021, 50 novel molecular entities (NMEs) received FDA approval, out of which 23 were approved in the second half. On this note, let’s take a look at major drugs launched in the second half of 2021 (H2).  

Kerendia: Bayer: Chronic Kidney Disease associated with Type 2 Diabetes

In July, the US FDA approved Bayer’s Finerenone, the first non-steroidal, selective mineralocorticoid receptor (MR) antagonist under the brand name Kerendia. Finerenone 10 mg or 20 mg is indicated to reduce the risk of long-term eGFR decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with Type 2 Diabetes (T2D) and Chronic Kidney Disease (CKD).

CKD is a leading health problem all over the world and as per DelveInsight’s Chronic Kidney Disease Epidemiology Forecast Report, approx 100 million people are living with CKD in 7MM. According to the reports More than 160 million people worldwide suffer from CKD and T2D. Even when blood glucose and blood pressure levels of these patients are well-controlled, they are still at risk of Chronic Kidney Disease progression. This indicates that there is a significant unmet medical need for early intervention to prevent further end-organ damage and premature death by slowing patients’ rate of decline in kidney function and lowering cardiovascular risk.

The FDA approval of finerenone was based on the positive results of the pivotal Phase III FIDELIO-DKD study, which was presented at the American Society of Nephrology’s (ASN) Kidney Week Reimagined 2020 and simultaneously published in the New England Journal of Medicine in October 2020, and follows the FDA’s Priority Review designation in January 2021.

The FIDELIO-DKD Phase III trial is the first large contemporary positive outcomes study in patients with Chronic Kidney Disease and Type 2 Diabetes with a primary composite endpoint consisting solely of kidney-specific outcomes. It is also a component of the largest global Phase III clinical trial program in CKD and T2D to date.

Finerenone has also been submitted for marketing authorization in the European Union (EU), China, and a number of other countries around the world, and these applications are currently being reviewed.

Bylvay: Albireo Pharma: Pruritus

Bylvay (odevixibat) is the first drug approved in the United States for Pruritus treatment in all subtypes of Progressive Familial Intrahepatic Cholestasis (PFIC). Bylvay is a non-systemic, once-daily ileal bile acid transport inhibitor that acts locally in the small intestine.

Bylvay manufactured by Albireo Pharma received FDA approval in July. The FDA approval of Bylvay was supported by data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted in PFIC. Bylvay met both its primary endpoints of pruritus (p=0.004) and serum bile acid (p=0.003) in PEDFIC 1, a randomized, double-blind, placebo-controlled study, and was well tolerated, with a very low incidence of diarrhea/frequent bowel movements (9.5 percent of treated patients vs. 5.0 percent of placebo patients). PEDFIC 2, a long-term, open-label Phase 3 extension study, found that Bylvay delivered sustained reductions in serum bile acids as well as improvements in pruritus assessments, growth, and other liver function markers in patients treated for up to 48 weeks. Bylvay was well tolerated in both studies, with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. There were no serious side effects associated with the treatment.

Albireo launched Bylvay immediately in order to help patients and families affected by PFIC. Bylvay is recommended to be taken once daily in the morning with a meal at a dose of 40 mcg/kg. If the clinical response is insufficient after three months, the dosage may be increased in 40 mcg/kg increments up to 120 mcg/kg once daily, not to exceed a total daily dose of 6 mg. The medication is only available with a prescription, and treatment should be initiated and overseen by a doctor with experience in the management of PFIC.

With the FDA approval in the United States, the FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV), which the Company intends to monetize. The Company had USD 186.3 million in cash and cash equivalents as of June 30, 2021 (unaudited) and anticipates an operating cash burn of USD 130-135 million in 2021. Excluding any proceeds from the planned sale of the PRV, the Company believes that its cash and cash equivalents will cover its operating and capital expenditure needs until 2023, which should be enough to launch Bylvay and expand beyond PFIC.

Albireo is also researching the use of Bylvay in other rare pediatric Cholestatic Liver Diseases, such as Biliary Atresia and Alagille syndrome, through the BOLD Phase 3 clinical trial in patients with Biliary Atresia and the ASSERT Phase 3 clinical trial in Alagille syndrome. The ASSERT trial’s topline data is expected in 2022, and the BOLD trial’s data is expected in 2024.

Nexviazyme: Sanofi: Late-Onset Pompe Disease

Nexviazyme (avalglucosidase alfa-ngpt) has been approved by the US Food and Drug Administration (FDA) in August for the treatment of patients one year of age and older with late-onset Pompe Disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe. Nexviazyme developed by Sanofi is an enzyme replacement therapy (ERT) that is specifically designed to target the mannose-6-phosphate (M6P) receptor, which is the key pathway for enzyme replacement therapy cellular uptake in Pompe Disease. Nexviazyme has been shown in clinical trials to improve respiratory function and walking distance in patients.

In the United States, an estimated 3,500 people suffer from Pompe disease, which can manifest as infantile-onset Pompe Disease (IOPD), the most severe form of Pompe Disease with a rapid onset in infancy, or late-onset Pompe Disease (LOPD), which gradually damages muscles over time.

Nexviazyme has shown to be beneficial for people suffering from late-onset Pompe disease. Nexviazyme demonstrated improvements in respiratory function and walking distance measures in people with LOPD in the pivotal Phase 3 trial (COMET), establishing its safety profile. Patients treated with Nexviazyme had a 2.9-point improvement (SE=0.9) in forced vital capacity (FVC) percent-predicted at Week 49, the study’s primary endpoint. At Week 49, patients treated with Nexviazyme had a 2.4-point greater improvement in FVC percent-predicted compared to patients treated with alglucosidase alfa, meeting the non-inferiority criterion (p=0.0074; 95 percent CI, -0.13, 4.99). Nexviazyme’s statistical superiority over alglucosidase alfa was not achieved (p=0.06).

Nexviazyme is given as a monotherapy ERT every two weeks. The recommended dose is based on body weight (20 mg/kg for LOPD patients 30 kg or 40 mg/kg for LOPD patients 30 kg) and is administered incrementally via intravenous infusion.

Furthermore, several clinical trials in Pompe Disease are ongoing by leading companies including Genzyme, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others to improve the  Pompe Disease treatment outlook.

Nexviazyme previously received FDA Breakthrough Therapy and Fast Track designations for Pompe Disease treatment. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its approval to avalglucosidase alfa. While Sanofi is pleased that the CHMP recognized the clinically meaningful improvements demonstrated in the avalglucosidase alfa development program, the CHMP also concluded that avalglucosidase alfa does not qualify as a New Active Substance (NAS). As a result, Sanofi has requested that the CHMP opinion on the NAS conclusion be re-examined. In addition, Sanofi filed avalglucosidase alfa in Japan in January 2021. Avalglucosidase alfa’s safety and efficacy have not been thoroughly evaluated by any regulatory authority outside of the United States.

Exkivity: Takeda: NSCLC

Exkivity (mobocertinib) has been approved by FDA in August for the treatment of adult patients with locally advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) who have Epidermal Growth Factor Receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test and whose disease has progressed on or after platinum-based chemotherapy. Exkivity, the first and only FDA-approved oral therapy specifically designed to target EGFR Exon20 insertion mutations, was granted priority review and received Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation. This indication has been granted Accelerated Approval based on the overall response rate (ORR) and DoR. The continuation of approval for this indication may be conditional on the verification and description of clinical benefit in a confirmatory trial.

Concurrently, the FDA approved Thermo Fisher Scientific’s Oncomine Dx Target Test as an NGS companion diagnostic for Exkivity to identify NSCLC patients with EGFR Exon20 insertions. NGS testing is critical for these patients because it allows for more precise diagnoses than polymerase chain reaction (PCR) testing, which detects less than half of EGFR Exon20 insertions.

The FDA approval is based on the results of the platinum-pretreated population in the EXKIVITY Phase 1/2 trial, which included 114 patients with EGFR Exon20 insertion+ NSCLC who had previously received platinum-based therapy and were treated at the 160 mg dose. The Phase 1/2 trial results were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and demonstrated a confirmed ORR of 28 percent per independent review committee (IRC) (35 percent per investigator), as well as a median DoR of 17.5 months per IRC, a median OS of 24 months, and a median PFS of 7.3 months per IRC. 

As per DelveInsight’s analysis, the current approval is for Refractory Patient pool but it is expected to get approval in First-line as well in couple of years and may generate a revenue of more than USD 500 million in 7MM despite competition from amivantamab-vmjw, another approval for the same patient segment by J&J during the same time frame

~ Rajesh Kumar, Functional Head, Forecasting

Find out How is Rybrevant giving tough competition to ExKivity?

Livmarli: Mirum Pharmaceuticals: Cholestatic Pruritus with Alagille Syndrome

The FDA has approved Livmarli (maralixibat) oral solution for Cholestatic Pruritus Treatment in patients one year of age and older with Alagille Syndrome (ALGS) in September. Livmarli developed by Mirum Pharmaceuticals, is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, is the first and only FDA-approved medication for this rare liver disease that affects approximately 2,000 to 2,500 children in the United States. Livmarli is now on the market and can be prescribed. Mirum also received a rare pediatric disease priority review voucher in conjunction with the approval.

There have been no approved treatments for Cholestatic Pruritus in Alagille Syndrome to date, and many children eventually require major surgical interventions such as liver transplantation for refractory pruritus. The FDA approval of Livmarli represents a significant shift in the Alagille syndrome treatment paradigm and provides hope to the many families who have suffered from the persistent itch for far too long.

Livmarli was approved based on the pivotal ICONIC study, as well as five years of data from supportive studies, which resulted in a strong body of evidence in 86 patients with ALGS. Data from ICONIC showed statistically significant reductions in pruritus, one of the most common and difficult symptoms of the disease, that lasted four years.

Livmarli is currently being studied in late-stage clinical trials for other rare cholestatic liver diseases, such as progressive familial intrahepatic cholestasis and biliary atresia, both of which have been designated as Breakthrough Therapy and Orphan Drug.

Scemblix: Novartis: Chronic Myeloid Leukemia

Scemblix (asciminib) developed by Novartis received the US Food and Drug Administration (FDA) approval in October for Chronic Myeloid Leukemia (CML) treatment in two different indications. Scemblix received accelerated approval from the FDA for adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) who had previously been treated with two or more tyrosine kinase inhibitors (TKIs) based on major molecular response (MMR) rate at 24 weeks, as well as full approval for adult patients with Ph+ CML-CP who had the T315I mutation. According to the Accelerated Approval Program, continued approval for the first indication may be contingent on clinical benefit verification and description from confirmatory evidence1. Scemblix is the first FDA-approved CML treatment that works by binding to the ABL myristoyl pocket, and it represents a significant step forward for patients who have developed resistance or intolerance to currently available TKI therapies. Scemblix, also known as a STAMP inhibitor in the scientific literature, is being studied in multiple treatment lines for CML-CP, including the ASC4FIRST Phase III study, which is evaluating Scemblix as a first-line treatment.

Current CML treatment may be limited for many patients due to intolerance or resistance, and sequential use of available TKIs is associated with increased failure rates. In a study of patients with CML who had previously received two TKIs, approximately 55% reported intolerance to the previous treatment. Furthermore, a pooled analysis in the second-line setting revealed that up to 70% of patients fail to achieve major molecular response (MMR) within two years of follow-up. Furthermore, patients who develop the T315I mutation are resistant to the majority of available TKIs, putting them at a higher risk of disease progression.

Scemblix was approved by the FDA based on findings from the Phase III ASCEMBL trial and a Phase I (NCT02081378) study involving patients with Ph+ CML-CP with the T315I mutation.

The ASCEMBL trial found that Scemblix nearly doubled the MMR rate compared to Bosulif (bosutinib) at 24 weeks (25 percent vs. 13 percent [P=0.029]) in patients with Ph+ CML-CP who had experienced resistance or intolerance to at least two TKIs. Patients in the Scemblix arm (n = 156) were more than three times more likely to discontinue treatment due to adverse reactions than patients in the Bosulif arm (n = 76) (7% vs. 25%). Upper respiratory tract infections and musculoskeletal pain; a decrease in platelet and neutrophil counts, a decrease in hemoglobin, an increase in triglycerides, creatine kinase, and alanine aminotransferase; and a decrease in triglycerides, creatine kinase, and alanine aminotransfer (ALT) were the most common (incidence 20%) adverse reactions and laboratory abnormalities in the Scemblix arm, respectively.

Besremi: AOP Orphan: Polycythaemia Vera

Besremi (ropeginterferon alfa-2b-njft) injection has been approved by the FDA) in November to treat adults with polycythemia vera, a blood disease that causes an overproduction of red blood cells. It is a Rare Disease and as per Polycythemia Vera – Market and Epidemiology report approximately 6,000 Americans are diagnosed with polycythemia vera every year. Approval of Besremi is a light of hope for the patients suffering from PV asBesremi is the first FDA-approved medication for polycythemia vera that patients can take regardless of their treatment history, as well as the first interferon therapy approved specifically for polycythemia vera treatment. Besremi was designated as an orphan drug for this indication.

The FDA approval was based on the efficacy findings of the PEGINVERA clinical study program. After 7.5 years of treatment with ropeginterferon alfa-2b-njft, 80% of polycythemia vera patients achieved a hematologic response, with 61 percent achieving a complete hematologic response.

Besremi, on the other hand, can cause elevated liver enzymes, low levels of white blood cells, low levels of platelets, joint pain, fatigue, itching, upper airway infection, muscle pain, and flu-like illness. Urinary tract infection, depression, and transient ischemic attacks are all possible side effects. Furthermore, due to the risk of fetal harm, people who may be pregnant should be tested for pregnancy before using Besremi.

Livtencity: Takeda: Post-transplant Cytomegalovirus

Livtencity (Takeda) is a novel molecular entity that inhibits viral DNA replication, encapsidation, and nuclear egress by targeting CMV at pUL97. Livtencity (maribavir) has been approved by FDA in November for the treatment of adults and pediatric patients (12 years of age or older and weighing at least 35 kg) with post-transplant Cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir, or foscarnet. Livtencity is Takeda’s second new molecular entity to be approved by the FDA in the fiscal year 2021.

Discover more about the Major drugs launched in the first half of 2021

Livtencity was evaluated in the TAK-620-303 (SOLSTICE) trial, a global, multicenter, randomized, open-label, active-controlled superiority trial comparing the efficacy and safety of maribavir versus investigator-assigned treatment (IAT, conventional antiviral therapy) in 352 HSCT and SOT adult recipients with CMV infection refractory, with or without resistance, to one or a combination of conventional antiviral therapies: For up to 8 weeks, participants were randomly assigned to either maribavir (N=235) (400 mg, twice daily) or IAT (N=117) (as dosed by the investigator). Following the conclusion of the treatment period, subjects entered a 12-week follow-up period. The primary efficacy endpoint was CMV DNA level LLOQ (lower limit of quantification, [i.e. 137 IU/mL] as determined by COBAS® AmpliPrep/COBAS® TaqMan® CMV test at the end of Week 8).

Taste disturbance, nausea, diarrhea, vomiting, and fatigue were the most common adverse events occurring in >10% of patients receiving maribavir at all grades.

Prior to FDA approval, the FDA granted Livtencity (maribavir) Orphan Drug Designation for the treatment of clinically significant CMV viremia and disease in at-risk patients, as well as Breakthrough Therapy Designation for CMV infection treatment and disease in transplant patients who were resistant or refractory to prior therapy. Takeda is excited to continue discussions with regulatory agencies around the world in order to potentially bring maribavir to patients worldwide. In an ongoing Phase 3 clinical trial, the company is also looking into maribavir as a first-line CMV treatment in hematopoietic stem cell transplant recipients.

Cytalux: On Target Laboratories: Ovarian Cancer

The Food and Drug Administration (FDA) in the United States has approved Cytalux as an adjunct for the intraoperative identification of malignant lesions in adult patients with ovarian cancer in November. Cytalux manufactured by On Target Laboratories is the first targeted fluorescent imaging agent to illuminate ovarian cancer intraoperatively, allowing for the detection and removal of more cancer. Cytalux, which can be given intravenously as early as one hour before surgery, binds to folate receptors that are overexpressed in most epithelial ovarian cancers and illuminate the surgical site with near-infrared light. 

The FDA approval of Cytalux was based on data from a Phase 3 registration trial, which showed that Cytalux identified additional lesions that would have been missed in 27% of patients (N=134, 95 percent CI [0.196, 0.352]). In a subgroup analysis of patients with confirmed ovarian cancer who underwent interval debulking surgery after chemotherapy, the rate was 40% (N=58, 95 percent CI [0.270, 0.534]). This subgroup analysis used a smaller data set than the primary endpoint and was not adjusted for error, so the results may be exaggerated and should be interpreted with caution.

The FDA granted Cytalux Priority Review, Fast Track, and Orphan designations. Furthermore, Cytalux is being studied in Phase 3 ELUCIDATE trial for lung cancer under Fast Track designation.

Vyvgart: argenx: Generalized Myasthenia Gravis

Vyvgart (efgartigimod alfa-fcab) by argenx received US Food and Drug Administration (FDA) approval in December for Generalized Myasthenia Gravis (gMG) treatment in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. These patients account for roughly 85% of the total gMG population. Vyvgart is a human IgG1 antibody fragment that binds to FcRn and reduces circulating immunoglobulin G (IgG) antibodies. AChR autoantibody action at the neuromuscular junction is a key driver of gMG. Vyvgart has achieved a regulatory milestone by becoming the first and only FDA-approved neonatal Fc receptor (FcRn) blocker.

Vyvgart was approved based on the findings of the global Phase 3 ADAPT trial, which was published in the July 2021 issue of The Lancet Neurology. The ADAPT trial met its primary endpoint, demonstrating that significantly more anti-AChR antibody-positive gMG patients were responders on the MG-ADL scale after Vygart treatment compared to placebo (68 percent vs. 30%; p0.0001).

Marketing Authorization Applications for efgartigimod for gMG treatment are currently being reviewed by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and the European Medicines Agency (EMA), with decisions from each agency expected in the first and second halves of 2022, respectively.

For the development and commercialization of efgartigimod in Greater China, argenx has an exclusive partnership agreement with Zai Lab. Zai Lab plans to apply for approval in Greater China by the middle of 2022. With the FDA approval of Vyvgart in the United States, argenx will receive a USD 25 million milestone payment under the terms of the strategic agreement with Zai Lab. Furthermore, argenx has an exclusive agreement with Medison for the commercialization of efgartigimod in gMG in Israel. Medison plans to submit an application for approval in Israel in the second quarter of 2022.

Tezspire: Amgen/AstraZeneca: Asthma

Tezspire is a first-in-class biologic for severe asthma that targets thymic stromal lymphopoietin (TSLP), an epithelial cytokine, at the top of the inflammatory cascade. Tezspire (tezepelumab-ekko) by Amgen and AstraZeneca has been approved by the FDA in December for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.

Tezspire was approved by the FDA after a Priority Review and based on the results of the PATHFINDER clinical trial program. The application included data from the pivotal NAVIGATOR Phase 3 trial, in which Tezspire outperformed placebo in every primary and key secondary endpoint in patients with severe asthma when added to standard therapy.

Tezspire is the first and only biologic for severe asthma that does not have an eosinophilic or allergic phenotype or biomarker limitation in its approved label. Tezspire is currently being evaluated by regulators in the EU, Japan, and a number of other countries around the world.

Leqvio: Novartis: HeFH and clinical ASCVD 

In December, the FDA approved Novartis’ Leqvio (inclisiran), the first and only small interfering RNA (siRNA) therapy to lower low-density lipoprotein cholesterol (LDL-C) with two doses per year after an initial dose and one at three months. 

In the United States, Leqvio is approved as an adjunct to diet and maximally tolerated statin therapy for adults with clinical atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) who require additional LDL-C lowering. Clinical trials are currently underway to investigate the effect of Leqvio on cardiovascular morbidity and mortality.

Leqvio lowers LDL-C levels in the bloodstream by improving the liver’s natural ability to prevent the production of a protein that contributes to high circulating cholesterol levels. It is a subcutaneous injection administered by a healthcare provider with an initial dose, three months later, and then every six months1. This approach may benefit those who have difficulty sticking to self-administered medications with higher dosing frequency. Leqvio is expected to be available in early January 2022.

The FDA granted approval based on the findings of the large-scale Phase III ORION-9, -10, and -11 clinical trials, in which all 3,457 participants with ASCVD or HeFH had elevated LDL-C while receiving a maximally tolerated dose of statin therapy. At month 17, Leqvio delivered effective and sustained LDL-C reductions of up to 52% vs. placebo in Phase III trials and was reported to be well-tolerated with a safety profile comparable to placebo. Mild to moderate injection site reaction (including pain, redness, and rash), joint pain, urinary tract infection, diarrhea, chest cold, pain in legs or arms, and shortness of breath were the most common side effects.

Under a license and collaboration agreement with Alnylam Pharmaceuticals, a leader in RNAi therapeutics, Novartis has obtained global rights to develop, manufacture, and commercialize Leqvio.

Adbry: Leo Pharma: Moderate-to-Severe Atopic Dermatitis

Adbry (tralokinumab) developed by Leo Pharma has been approved by the US Food and Drug Administration (FDA) in December for Moderate-to-Severe Atopic Dermatitis treatment in adults 18 years of age and older whose disease is not adequately controlled with topical prescription therapies or when those therapies are contraindicated. Adbry can be used in conjunction with or without topical corticosteroids. Adbry (tralokinumab) is a human monoclonal antibody and the first and only FDA-approved biologic that specifically binds to and inhibits the IL-13 cytokine, which is a key driver of the signs and symptoms of Atopic Dermatitis.

Adtralza was already approved by the European Commission for adults with moderate-to-severe AD in Europe and by the Medicines and Healthcare products Regulatory Agency in Great Britain, in June 2021. The drug was launched in the UK in August 2021. Due to significant capacity challenges, NICE has paused the development of the Single Technology Appraisals for abrocitinib for treating moderate to severe AD in people aged 12 and over, upadacitinib for treating moderate to severe AD in people aged 12 and over and tralokinumab for treating moderate to severe AD. All three topics will be appraised together as a Multiple Technology Appraisal and the expected publication date is 17 October 2022.

~ Sadaf Javed, Assistant Project Manager

Adbry was approved based on the safety and efficacy results of the ECZTRA 1, 2, and 3 pivotal Phase 3 trials, which included nearly 2,000 adult patients with Moderate-to-Severe Atopic Dermatitis. 1 Safety data were analyzed from a pool of five randomized, double-blind, placebo-controlled trials, including ECZTRA 1, 2, and 3, as well as a dose-finding trial and a vaccine response trial.

Adbry will be available in a 150 mg/mL prefilled syringe for subcutaneous injection, with a 600 mg starting dose followed by 300 mg every other week. Adbry can be used in conjunction with or without TCS. For patients weighing less than 100 kg who have clear or nearly clear skin after 16 weeks of treatment, a dose of 300 mg every four weeks may be considered.

LEO Pharma will launch the Adbry Advocate Program to help eligible patients gain access to Adbry. This program will support U.S. patients during their diagnosis and treatment with Adbry.

We expect Adbry (Tralokinumab) to compete with the Regeneron/Sanofi’s Dupixent, the existing blockbuster interleukin therapy for moderate to severe AD in Adults and Adolescents.  Other competitor Interleukins in the AD pipeline are Eli LiLLy’s Lebrikizumab and Galderma’s Nemolizumab, which are also expected to launch in the year 2022 and 2023 respectively in the United States. Moreover, as per our estimates, we expect Adbry to reach a market size of approximately USD 1,100 Million in the United States and approximately USD 900 Million in the EU5 countries combined by the year 2030.

~ Sadaf Javed, Assistant Project Manager

The FDA approval is the fifth global regulatory approval for tralokinumab in 2021. Tralokinumab is marketed outside of the United States under the brand name Adtralza and is currently approved in the European Union, the United Kingdom, Canada, and the United Arab Emirates.

What’s Ahead?

During the first half of 2021, the US FDA approved a large number of drugs as compared to the second half. Though in the second half, FDA has granted many orphan drug designations, breakthrough treatment designations, and biologics licensing applications. Among these are Sanofi’s Nexviazyme for late-onset Pompe Disease, Takeda’s Livtencity for post-transplant Cytomegalovirus, On Target Laboratories’ Cytalux for Ovarian Cancer, and others. Many leading players in the pharma market have launched their key assets in the second half which have the potential to become blockbuster drugs. Unlike the first half, we haven’t witnessed many drugs from the oncological sector. But as we have moved into 2022, we may see some key drug launches this year especially from the oncological sector as the outlook of the oncological sector is looking promising. 


1. What are the top drugs launched in 2021?

The top drugs launched in the second half of 2021 include Kerendia, Bylvay, Nexviazyme, Exkivity, Livmarli, Scemblix, Besremi, Livtencity, Cytalux, Vyvgart, Tezpire, Leqvio, Adbry, and others.

2. Which leading players launched their key assets in 2021?

The leading players that launched their key products in the second half of 2021 include Bayer, Albireo Pharma, Sanofi, Takeda, Mirum Pharmaceuticals, Novartis, AOP Orphan, On Target Laboratories, argenx, Amgen, AstraZeneca, Leo Pharma, and others.

3. How many companies are developing therapies for pompe disease?

Several companies such as Genzyme, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others are also developing novel therapies for Pompe Disease treatment.