CAPR News & Events

Dr. Marty Makary, the commissioner of the Food and Drug Administration, resigned on Tuesday, according to multiple media reports citing administration officials. Makary ultimately resigned over concerns about the administration's decision to authorize fruit-flavored e-cigarettes, which he opposed, four people familiar with the matter told The New York Times. Kyle Diamantas, the F.D.A.'s top food regulator, was named the acting commissioner, according to The Times and Politico, which was first to report of Makary's resignation. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks that could be impacted by the news include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

Capricor Therapeutics (CAPR) announced that it has filed a lawsuit against Nippon Shinyaku (NPNKF) and its U.S. subsidiary, NS Pharma, over the parties' U.S. distribution agreement for Deramiocel, Capricor's investigational cell therapy for the treatment of Duchenne muscular dystrophy. "Deramiocel represents one of the most significant therapeutic advances for DMD, addressing both its skeletal and cardiac manifestations. NS Pharma's inaction may now jeopardize patients' access to this life-changing treatment," the company said. Capricor's complaint details how "a fundamental pricing flaw in the Commercialization and Distribution Agreement with NS Pharma will prevent patients covered by Medicare, Medicaid, or private insurance from accessing the therapy. Capricor has sought to work in good faith to fix this pricing mechanism with NS Pharma, but NS Pharma has refused to compromise. NS Pharma also has failed to adequately prepare for commercial launch of Deramiocel, and Capricor is now taking legal action to ensure there is a path for Deramiocel to reach the patients who urgently need it." The U.S. Food and Drug Administration has granted Deramiocel Priority Review, with a target PDUFA action date of August 22, 2026. The FDA review process and expected timing remain unchanged.