Company: Fresenius Kabi and Formycon

Drug: Otulfi

Indication: Crohn's disease, ulcerative colitis, plaque psoriasis, and active psoriatic arthritis

The FDA has approved Otulfi, a new biosimilar to Stelara (ustekinumab), from partners Fresenius Kabi and Formycon for the treatment of Crohn's disease, ulcerative colitis, moderate to severe plaque psoriasis, and active psoriatic arthritis. According to the two companies, the FDA granted final approval for Otulfi’s subcutaneous and intravenous formulations after a comprehensive assessment of an integrated data package that included analyses, preclinical, clinical, and manufacturing data. Fresenius Kabi is permitted to begin marketing Otulfi by February 22, 2025, marking it as the fourth biosimilar approved in the U.S.

Company: Sanofi and Regeneron

Drug: Dupixent

Indication: Chronic obstructive pulmonary disease (COPD)

Sanofi and Regeneron's flagship drug Dupixent has won its seventh indication and becomes the first biologic to treat chronic obstructive pulmonary disease (COPD) in the U.S. The companies supported their application for Dupixent to treat COPD with data from two Phase III trials, which showed that the injection reduced the annualized rate of moderate or severe COPD exacerbations by 30% and 34% compared to placebo. Dupixent also statistically improved forced expiratory volume levels after 12 weeks post-bronchodilator, reaching the trial’s secondary endpoint. The safety profile is consistent with Dupixent's use in other approved indications, including asthma and eczema.

Company: Bristol Myers Squibb (BMS)

Drug: Cobenfy

Indication: Schizophrenia

BMS made history with the approval of Cobenfy (formerly KarXT), the first new treatment for schizophrenia in 35 years. While schizophrenia treatments have focused primarily on blocking dopamine signaling throughout the brain, Cobenfy targets M1 and M4 muscarinic receptors. The approval is supported by data from three registration trials, where the drug demonstrated benefits in treating schizophrenia on both positive and negative symptom scales. The drug was initially developed by Karuna Therapeutics, starting its development journey as an experimental treatment for Alzheimer's disease by Eli Lilly, which discovered its impact on psychotic symptoms of the disease. BMS chose the drug when it acquired Karuna in December 2023.

September 26

Company: AstraZeneca

Drug: Tagrisso

Indication: Non-small cell lung cancer (NSCLC)

AstraZeneca has expanded the indication for its NSCLC drug Tagrisso, as the FDA approved it for treating adults with unresectable Stage III epidermal growth factor receptor mutation (EGFRm) disease, which has not progressed during or after platinum-based chemotherapy. Tagrisso is particularly indicated for patients with exon 19 deletions or exon 21 mutations. The FDA's approval is supported by results from the Phase III LAURA trial, which showed that Tagrisso treatment reduced the risk of disease progression or death by 84% compared to placebo. AstraZeneca reports that Tagrisso also extended progression-free survival by 33.5 months compared to placebo. Dr. Suresh Ramalingam, executive director of Emory University Winship Cancer Institute and the trial's principal investigator, called this approval a “significant breakthrough” for this patient population. Tagrisso is also approved for first-line treatment of metastatic EGFR mutation NSCLC as a monotherapy and in combination with chemotherapy, as well as for adjuvant treatment in early disease.

Company: IntraBio

Drug: Aqneursa

Indication: Type C Niemann-Pick disease

Just days after the FDA approved Zevra Therapeutics’ Miplyffa for this extremely rare neurodegenerative disease, the FDA granted the green light for IntraBio’s Aqneursa. This drug is specifically designed to alleviate neurological symptoms in adults and children with Type C Niemann-Pick disease (NPC) who weigh at least 15 kg. The approval of Aqneursa is supported by data from a randomized, double-blind, placebo-controlled crossover study. During a 12-week period, participants showed better functional performance based on assessments of gait, sitting, posture, and speech. Aqneursa was well tolerated, with common side effects including vomiting, abdominal pain, difficulty swallowing, and upper respiratory infections. Janet Maynard, director of the FDA’s Office of Rare Diseases, Pediatrics, Urology, and Reproductive Medicine, stated that the second NPC approval within a week “highlights” the FDA's commitment to supporting the development of innovative therapies for rare diseases.

Company: UCB

Drug: Bimzelx

Indication: Psoriatic arthritis, non-radiographic axial spondyloarthritis, and ankylosing spondylitis

UCB's Bimzelx has been approved for treating three chronic immune-mediated inflammatory diseases: psoriatic arthritis (PsA), non-radiographic axial spondyloarthritis (nr-axSpA), and ankylosing spondylitis (AS). According to UCB, Bimzelx is the first approved drug for all three indications, with a mechanism of action as a selective inhibitor of two key inflammatory-driving cytokines, interleukin 17A (IL-17A) and interleukin 17F (IL-17F). The approval of Bimzelx for active PsA is supported by two Phase III studies, which showed statistically significant improvements in joint and skin symptoms at 16 weeks compared to placebo, with results sustained up to 52 weeks. Additionally, in two Phase III studies for nr-axSpA and AS, Bimzelx demonstrated statistically significant improvements in signs and symptoms at 16 weeks compared to placebo, with results lasting up to one year. Bimzelx was previously approved by the FDA in October 2023 for adult patients with plaque psoriasis suitable for systemic therapy or phototherapy.

Company: Zevra Therapeutics

Drug: Miplyffa

Indication: Type C Niemann-Pick disease

The FDA has approved Zevra Therapeutics’ Miplyffa for treating the ultra-rare progressive neurodegenerative disease Type C Niemann-Pick disease (NPC). Specifically, Miplyffa is authorized for use in conjunction with miglustat for treating adults and children aged 2 years and older with NPC. The approval of Miplyffa is supported by data from a 12-month multicenter, randomized, double-blind, placebo-controlled trial investigating treatment in patients aged 2 to 19 with NPC. The trial's results indicated that Miplyffa combined with miglustat prevented disease progression over 12 months, as evidenced by a 0.2-point decrease in the re-scored four-domain NPC clinical severity scale compared to baseline. In contrast, patients receiving miglustat alone had a progression score of 1.9 points.

September 20

Company: AstraZeneca

Drug: FluMist

Indication: Influenza

The FDA has approved the first self-administered influenza vaccine. FluMist is a needle-free nasal spray authorized for use by adults under 49 years of age and by parents or caregivers for children aged 2 to 17. Availability studies have shown that the intended population can self-administer or vaccinate.

Company: Johnson & Johnson

Drug: Rybrevant

Indication: Non-small cell lung cancer

Johnson & Johnson has received its third approval for a non-small cell lung cancer (NSCLC) indication this year, with the FDA approving it for second-line treatment of locally advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon 19 deletions or L858R substitution mutations. Rybrevant is specifically authorized for patients with disease progression during or after treatment with EGFR tyrosine kinase inhibitors. This approval is supported by results from the Phase III MARIPOSA-2 trial, which showed that Rybrevant treatment reduced the risk of disease progression or death by 52% compared to chemotherapy alone. Patients receiving the combination regimen had a median progression-free survival of 6.3 months, compared to 4.2 months for those receiving chemotherapy alone. The overall response rate for Rybrevant in combination with chemotherapy was 53%, compared to 29% for chemotherapy alone. In August, Rybrevant was approved in combination with lazertinib for locally advanced or metastatic NSCLC with EGFR exon 19 deletions or L858R substitutions. Prior to that, Rybrevant in combination with chemotherapy was approved in March for first-line treatment of locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations.

Company: AstraZeneca

Drug: Fasenra

Indication: Eosinophilic granulomatosis with polyangiitis (EGPA)

The FDA has approved Fasenra for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), a rare immune-mediated disease that can lead to multi-organ failure and significant morbidity and mortality if not treated. The approval follows data from the Phase III, placebo-controlled, randomized trial, which showed that treatment with Fasenra significantly increased the proportion of patients who achieved remission of their disease compared to placebo. Fasenra is a monoclonal antibody that targets IL-5 to reduce eosinophil levels in the blood. The drug is already approved for patients with severe eosinophilic asthma, eosinophilic granulomatosis, and polyangiitis.

Company: PTC Therapeutics

Drug: Upstaza

Indication: A genetically defined form of amyotrophic lateral sclerosis (ALS)

PTC Therapeutics' Upstaza has been approved by the FDA for the treatment of a genetically defined form of amyotrophic lateral sclerosis (ALS) caused by a mutation in the SOD1 gene. Upstaza is a gene therapy designed to provide a working copy of the SOD1 gene to slow disease progression. The approval is based on a Phase II clinical trial demonstrating that Upstaza was able to reduce the levels of the toxic SOD1 protein in patients' blood and cerebrospinal fluid, leading to a significant improvement in survival and functional measures. Upstaza is the first gene therapy approved for ALS.

Company: Biogen and Eisai

Drug: Leqembi

Indication: Alzheimer's disease

Biogen and Eisai's Leqembi has gained FDA approval for treating patients with early Alzheimer’s disease based on positive results from the Phase III CLARITY AD trial. Leqembi is a monoclonal antibody designed to target amyloid-beta plaques in the brain. The trial showed a statistically significant slowing of cognitive decline in patients receiving the drug compared to placebo. The approval is a notable milestone in the treatment landscape of Alzheimer’s disease, which has faced numerous setbacks over the years. Biogen is launching Leqembi under the FDA's accelerated approval pathway, which allows for marketing based on early clinical trial evidence while requiring further studies to confirm clinical benefit. Leqembi's safety profile includes risks of infusion-related reactions and amyloid-related imaging abnormalities, which require close monitoring of patients.

Company: Amgen

Drug: A new formulation of Amgen's Repatha

Indication: Cardiovascular disease

Amgen has secured FDA approval for a new formulation of Repatha, the cholesterol-lowering injection, which now allows for self-administration every two weeks or once a month. The approval was based on results from a Phase III trial showing that the new formulation was effective at reducing low-density lipoprotein cholesterol levels in patients with high cardiovascular risk. Repatha is already approved as an adjunct to diet and exercise for reducing LDL cholesterol in adults with heterozygous familial hypercholesterolemia and in patients with atherosclerotic cardiovascular disease.