A patient’s death has prompted the FDA to place a partial clinical hold on Curis Inc.’s phase I/IIa study of emavusertib in leukemia. The patient, who had relapsed or refractory acute myeloid leukemia, experienced several conditions. One of them was rhabdomyolysis, a dose-limiting toxicity of emavusertib. Rhabdomyolysis is a breakdown of muscle fibers in the blood.
Opinion on Wall Street said the matter could have gone either way, but in the end Akebia Therapeutics Inc.’s vadadustat, a HIF prolyl-hydroxylase inhibitor for anemia caused by chronic kidney disease (CKD), garnered a complete response letter (CRL) instead of approval from the FDA. The news slammed Akebia shares (NASDAQ:AKBA), which closed at 82 cents, down $1.61, or 66%. Specifically, the agency said that the data in the NDA do not support a favorable benefit-risk assessment of vadadustat in dialysis-dependent (DD) and non-DDs patients.
Unless the U.S. FDA once again overrides its Peripheral and Central Nervous System Drugs Advisory Committee, it looks like the ALS community will have a longer wait for an additional tool against the fatal, degenerative disease. After hearing from both Amylyx Pharmaceuticals Inc. and FDA reviewers, along with 26 people testifying during the open public hearing, the committee voted March 30 against approval of AMX-0035, a fixed-dose combination of sodium phenylbutyrate and taurursodiol, as a much-needed treatment for amyotrophic lateral sclerosis (ALS).
Zogenix Inc.’s Fintepla (fenfluramine) cleared the U.S. FDA hurdle shortly after its March 25 PDUFA date, expanding the drug’s use in patients with Lennox-Gastaut syndrome (LGS) and validating UCB SA’s $1.9 billion acquisition of Emeryville, Calif.-based firm, which closed earlier this month.
Investors didn’t respond well to the U.S. FDA’s briefing document for the March 30 advisory committee meeting on Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) candidate. Shortly after the materials for the adcom were posted Monday, Amylyx (NASDAQ:AMLX) dropped from a morning high of $25.68 per share to an all-time low of $10.49 in the heaviest trading since the company went public in January. With share volume exceeding 15 million, Amylyx rebounded somewhat, ending the day at $16.01, down nearly 36% from its March 25 close of $25.
The beleaguered PI3K-delta inhibitor space took another blow after MEI Pharma Inc. and partner Kyowa Kirin Co. Ltd. said the U.S. FDA won’t greenlight zandelisib without data from a randomized study. The firms had hoped to win accelerated approval in relapsed/refractory follicular lymphoma and marginal zone lymphoma based on a single-arm phase II study called Tidal.
Innovent Biologics Co. Ltd. and Eli Lilly and Co. are "assessing next steps" for their jointly developed PD-1 inhibitor, sintilimab, following receipt of a complete response letter (CRL) from the U.S. FDA. The pair sought approval of a BLA for sintilimab plus pemetrexed and platinum chemotherapy for the first-line treatment of people with nonsquamous non-small-cell lung cancer (NSCLC) but found near-unanimous opposition from FDA advisers dissatisfied with China-only trial data submitted in support of the application. The medicine is already approved for multiple indications in China, where it’s marketed as Tyvyt.
The U.S. FDA has approved Novartis AG’s Pluvicto (lutetium Lu 177 vipivotide tetraxetan, formerly referred to as 177Lu-PSMA-617) for treating adults with metastatic prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer. The treatment is the indication’s first FDA-approved targeted radioligand therapy that contains a radioisotope.
As congressional scrutiny of the U.S. FDA’s accelerated approval path continues, the agency is focusing research efforts into appropriate disclosure on direct-to-consumer websites about a drug’s accelerated approval and the status of confirmatory trials. Previous research by the FDA’s Office of Prescription Drug Promotion (OPDP) found that 27% of DTC websites providing information about a drug with accelerated approval don’t disclose that the products are on the market through accelerated approval.
Citing a court order for its haste, the U.S. FDA skipped the draft and went straight to issuing a final guidance that will change how certain ophthalmic drugs are regulated.
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