FDA Action Alert: BioXcel (April 5), Alnylam and Merck
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After wrapping up March with a flurry of PDUFA dates, the U.S. Food and Drug Administration has a more leisurely calendar for the first three weeks of April, at least in terms of drug approvals. Read on for details.
BioXcel’s Schizophrenia and Bipolar Disorders Drug
BioXcel Therapeutics, based in New Haven, Conn., has a target action date of April 5, 2022, for its New Drug Application (NDA) for BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II. It originally had a PDUFA date of January 5, but the agency requested additional information related to analyses of some of the clinical data. The FDA indicated it would require additional time to review.
BXCL501 is an orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2a receptor agonist. The submission is based on data from the SERENITY I trial for schizophrenia-related agitation, and SERENITY II for bipolar disorder-related agitation, and dementia-related agitation (TRANQUILITY). The drug has received Breakthrough Therapy designation for acute treatment of agitation associated with dementia and Fast Track designation for agitation associated with schizophrenia, bipolar disorders and dementia.
In BioXcel’s 2021 financial report on March 10, Vimal Mehta, Ph.D., chief executive officer of BioXcel, noted, “As we near potential approval, we remain focused on executing our launch readiness plan.”
Alnylam’s Vutrisiran for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
Alnylam Pharmaceuticals, headquartered in Cambridge, Massachusetts., has a target action date of April 14 for its NDA for vutrisiran. The RNA interference therapy is being evaluated for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It has been granted Orphan Drug Designation in the U.S. and European Union, as well as Fast-Track designation in the U.S. hATTR amyloidosis is an inherited, progressively debilitating and fatal illness caused by mutations in the TTR gene. The TTR protein is mostly generated in the liver and typically is a carrier of vitamin A. Damage to the gene causes abnormal myeloid protein to accumulate, which damages organs and tissues, such as the nerves and heart.
The NDA is partially built on results from the Phase III HELIOS-A study and APOLLO studies. In the HELIOS-A study, at month 18, the drug demonstrated improvements in exploratory cardiac endpoints, some echocardiographic parameters, and technetium uptake in the heart, suggestive of reduced cardiac amyloid burden.
Merck’s Zerbaxa for Pediatric Complicated Urinary Tract Infections
Merck & Co.has a target action date of April 21 for its supplemental NDA (sNDA) for Zerbaxa (ceftolozane and tazobactam) to treat pediatric complicated urinary tract infections. It also has a target action date of May 2 for the same drug for complicated intra-abdominal infections.
Zerbaxa is an antibacterial combination drug for intravenous infusion, made up of the cephalosporin antibacterial drug ceftolozane sulfate and the beta-lactamase inhibitor tazobactam sodium. It is indicated for the treatment of adults with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP), caused by several susceptible Gram-negative microorganisms: Enterobacter cloacae, E. coli, Haemophilus influenzae, Klebsiella oxytoca, Klebsiella pneumoniae, Proteus mirabilis, Pseudomonas aeruginosa and Serratia marcescens.