FDA Action Alert: A Very Busy Week for Drug Application Decisions

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The U.S. Food and Drug Administration has a very busy week ending the month of March and moving into April. Here’s a look.

Bristol Myers Squibb’s Reblozyl for Anemia in Beta Thalassemia Patients

Bristol Myers Squibb had a target action date of March 27, 2022, for its supplemental Biologics License Application (sBLA) for Reblozyl (luspatercept-aamt) for anemia in adults with non-transfusion dependent (NTD) beta thalassemia. The agency requested additional data, which BMS supplied, and the FDA called it a major amendment and added three months to the target action date. The PDUFA date is now June 27. 

The drug is a first-in-class erythroid maturation agent. It is being jointly developed by BMS and Merck via Merck’s acquisition of Acceleron. It is already approved in the U.S. for anemia in adults with beta thalassemia who require regular RBC transfusions. It is also approved for anemia failing an erythropoiesis-stimulating agent and requires two or more RBC units over eight weeks in adults with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

Antares’ Tlando for Testosterone Replacement Therapy

Antares Pharma and Lipocine have a target action date of March 28 for its resubmission for its New Drug Application (NDA) for Tlando (testosterone undecanoate), an oral treatment for testosterone replacement therapy. It was granted tentative approval from the FDA as a twice-daily oral formulation of testosterone for testosterone replacement therapy for conditions associated with a deficiency or absence of endogenous testosterone or hypogonadism in adult males. The agency concluded the drug met all required efficacy, quality and safety standards needed for approval and will be eligible for final approval and marketed in the U.S. when the exclusivity period previously granted to Clarus Therapeutics for Jatenzo expires on March 27.

Merck’s Keytruda for Advanced Endometrial Carcinoma

Merck had a target action date of March 28 for its sBLA for Keytruda (pembrolizumab), its anti-PD-1 checkpoint inhibitor, as a monotherapy for patients with advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression after previous systemic treatment in any setting and are not candidates for curative surgery or radiation. Keytruda is already approved for adults and children with unresectable or metastatic MSI-H or dMMR solid tumors that have progressed after previous treatment and have no satisfactory alternative treatment options. It was approved for this new indication on March 21.

Akebia’s Vadadustat for Anemia Due to Chronic Kidney Disease

Akebia Therapeutics has a target action date of March 29 for its NDA for vadadustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor for anemia due to chronic kidney disease in adults on dialysis and not on dialysis. It is being developed with Otsuka Pharmaceutical Co.

In a June 1, 2021 statement, John P. Butler, Akebia’s chief executive officer, stated, “The acceptance of our vadadustat NDA filing marks another important milestone for Akebia and Otsuka, as we work to bring a new oral treatment option to patients living with anemia due to CKD.”

Amylyx’s ALS Drug to Be Reviewed by Advisory Committee

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee is holding a meeting on March 30 to discuss Amylyx Pharmaceuticals’ NDA for sodium phenylbutyrate/taurursodiol powder for oral suspension for the treatment of amyotrophic lateral sclerosis (ALS). The drug has been granted Priority Review.

“There are few treatments approved for ALS, a devastating disease that impacts a person’s ability to move, speak, eat and breathe,” said Dr. Lahar Mehta, Head of Global Clinical Development of Amylyx,” in February 2022. “We look forward to a robust scientific discussion with the members of the advisory committee panel regarding the clinical data submitted to support our New Drug Application for AMX0035.”

Gilead/Kite’s Yescarta for Second-Line R/R Large B-Cell Lymphoma

Kite, a Gilead Company, has a target action date of April 1 for its sBLA under Priority Review for Yescarta, its CAR T-cell therapy, for second-line relapsed or refractory large B-cell lymphoma (LBCL). The therapy was evaluated against the current standard of care in the Phase III ZUMA-7 study. With a median follow-up of two years, the trial hit the primary endpoint of event-free survival (EFS), demonstrating a 2.5-fold increase in patients who were alive at two years and did not require additional cancer treatment or experienced cancer progression, and a four-fold great median EFS compared to SOC.

Merck’s Vaxneuvance Vaccine Against Pneumococcal Disease in Children

Merck has a target action date of April 1 for its sBLA for Vaxneuvance (Pneumococcal 15-valent Conjugate Vaccine) for the prevention of invasive pneumococcal disease in children six weeks through 17 years of age. It has Priority Review status. The sBLA is supported by Phase II and III trials data in pediatric populations.

“Vaxneuvance has the potential to provide meaningful protection against invasive pneumococcal disease for children and infants by targeting pneumococcal strains, or serotypes, that contribute to substantial disease burden, including serotype 3, and broadening coverage to additional disease-causing serotypes, 22F and 33F, which are not included in the pneumococcal conjugate vaccine (PCV) currently available for this population,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories.

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