FDA Updates for Week of May 27: 2 Approvals including New Biosimilar


The FDA has made two approvals this week: the first interchangeable biosimilar of Soliris and another indication for Breyanzi for mantle cell lymphoma. The agency has delay a decision on Dupixent in COPD and has set goal dates for zolbetuximab in gastric cancer and zanidatamab in bile duct cancer.

FDA Approves First Interchangeable Biosimilar of Soliris

The FDA has approved Amgen’s Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab). Bkemv is approved for two of Soliris indications: to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and patients with atypical hemolytic uremic syndrome (aHUS).

PNH is an ultra-rare blood disorder that is characterized by the destruction of red blood cells. Atypical hemolytic uremic syndrome is a rare genetic disease in which tiny blood clots form in blood vessels and block blood flow. It can lead to kidney failure and heart disease.

An Amgen spokesperson said that Bkemve will launch no later later than March 2025 as part of its agreement with Alexion. A price will be available at that time, according to the spokesperson.

Bkemv is a monoclonal antibody that binds to the complement C5 protein and inhibits activation of the complement system, a part of the body's immune system. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.

Bkemv, like Soliris, has a boxed warning about the increased risk of serious and life-threatening meningococcal infections caused by Neisseria meningitidis, the bacteria that causes meningitis and other severe infections.

FDA Approves Breyanzi for Mantle Cell Lymphoma

The FDA has approved Breyanzi (lisocabtagene maraleucel; liso-cel) to treat adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor.

Mantle cell lymphoma is an aggressive, rare form of non-Hodgkin lymphoma (NHL), representing roughly 3% of all NHL cases.

Developed by Bristol Myers Squibb, Breyanzi is a CD19-directed chimeric antigen receptor (CAR) T cell therapy that is already approved to treat adult patients with large B-cell lymphoma; relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma; and relapsed or refractory follicular lymphoma.

The approval for mantle cell lymphoma is based on results from the MCL cohort of TRANSCEND NHL 001. In the 68 patients treated with Breyanzi and evaluated for efficacy, 85.3% responded to treatment, with 67.6% achieving a complete response.

FDA Sets Goal Date for Resubmitted Zolbetuximab in Gastric Cancer

The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of Nov. 9, 2024, for the resubmission of the biologics license application (BLA) for zolbetuximab. The agency is reviewing zolbetuximab as a first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma.

Developed by Astellas, zolbetuximab is first-in-class monoclonal antibody that targets and binds to Claudin 18.2 (CLDN18.2), a transmembrane protein.

In January 2024, the FDA issued a complete response letter (CRL) for the zolbetuximab application. The FDA indicated the CRL was because of unresolved deficiencies following its inspection of a third-party manufacturing facility. The BLA was resubmitted on May 9, 2024.

FDA Sets Date for Zanidatamab in Bile Duct Cancer

The FDA has granted priority review of Jazz Pharmaceuticals’ biologics license application (BLA) for zanidatamab to treat patients with unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC). The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of Nov. 29, 2024.

Biliary tract cancer is a group of rare and aggressive cancers. It develops in the bile ducts, from the liver to the small intestine

Zanidatamab is bispecific antibody that targets the human epidermal growth factor receptor 2 (HER2). Zanidatamab binds to two non-overlapping epitopes of the HER2 receptor, which blocks HER2 signaling and removes the HER2 protein from the cell surface. Zanidatamab is being developed by Jazz and BeiGene, under license agreements from Zymeworks, which first developed the molecule.

Jazz’s submission is based on results from Cohort 1 of the phase 2b HERIZON-BTC-01 clinical. The trial demonstrated a primary endpoint of 41.3% confirmed objective response rate (cORR) with a median duration of response (DOR) of 12.9 months at a median follow-up of 12.4 months. Among the 33 responders, 49% had ongoing responses and 82% had a response lasting more than 16 weeks.

FDA Delays Decision for Dupixent in COPD

The FDA has extended by three months the target action date for the supplemental biologics license application (sBLA) for Dupixent (dupilumab) to treat patients with chronic obstructive pulmonary disease (COPD).

Developed by Sanofi and Regeneron Pharmaceuticals, Dupixent is being reviewed as an add-on maintenance treatment for uncontrolled COPD. The original Prescription Drug User Fee Act (PDUFA) date was June 27, 2024. The revised target action date is Sept. 27, 2024.

If approved, this would be the sixth indication for Dupixent and the only biologic therapy for COPD. Dupixent is also approved to treat eosinophilic esophagitis, moderate-to-severe atopic dermatitis, moderate-to-severe asthma, chronic rhinosinusitis with nasal polyposis and the rare skin disease prurigo nodularis.

The FDA has requested additional analyses on the efficacy of Dupixent in the two pivotal trials — BOREAS and NOTUS — which is considered a major amendment to the sBLA, Sanofi officials said in a news release.

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