In a Psychopharmacologic Drugs Advisory Committee, Otsuka Pharmaceutical Co. Ltd. and the U.S. FDA spent the morning discussing three clinical trials conducted as part of the supplemental NDA for Rexulti (brexpiprazole) combined with sertraline in treating adults with post-traumatic stress disorder (PTSD). One phase III study performed well and another phase III did not. A phase II study supporting Rexulti’s efficacy was included in the sNDA, but the FDA said it does not find the data convincing and wants more results. The committee will vote this later afternoon whether it thinks the available data showed efficacy when treating PTSD. The committee’s conclusions are not binding on the FDA, which will make its own decision whether to approve or not.

Third gene therapy death reported by Sarepta

Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) were trading at $18.05, down $3.92, or 17%, as Wall Street responded to word of a third death has occurred of a patient given the firm’s gene therapy. This time the drug was SRP-9004 and the indication was limb-girdle muscular dystrophy. Sarepta CEO Douglas Ingram said during a conference call this morning with analysts that the patient was a late-stage, non-ambulatory 51-year-old man participating in a phase I trial. He died about 11 weeks after dosing a month ago of liver failure, as did the two subjects who passed away after they were given Elevidys (delandistrogene moxeparvovec), the approved gene product for Duchenne muscular dystrophy.

Diamedica’s DM-199 shows promise for serious pregnancy disorder

After expanding development of DM-199 (rinvecalinase alfa) into preeclampsia last year, Diamedica Inc. rolled out early phase II results showing the recombinant form of human tissue kallikrein-1 reduced the mother’s blood pressure, did not cross the placental barrier and potentially improves blood flow to the fetus. A serious pregnancy disorder, preeclampsia develops after the 20th week of gestation with hallmark signs being high blood pressure and organ damage, including to the kidneys and liver. It affects up to 8% of pregnancies worldwide and can lead to stroke, placental abruption, eclampsia, premature delivery and death. Cathy Cluver, founder and leader of the Preeclampsia Research Unit at Tygerberg Hospital, Stellenbosch University, in South Africa where she is the principal investigator of the phase II open-label, single-arm, proof-of-concept trial, called DM-199’s ability to safely reduce blood pressure “an exciting development.”

EFPIA urges action to improve Europe’s clinical trials ecosystem

The pharmaceutical industry in Europe has taken the lead in pulling together a coalition of 17 industry groups, academics and clinicians to call for the urgent implementation of the EU Life Science Strategy published earlier this month, in order to rescue the clinical trial ecosystem from “a perilous situation.” The broad group of stakeholders was formed earlier this year to look at the chronic problems encountered in setting up and running clinical trials, and the European Federation for the Pharmaceutical Industry and Associations (EFPIA) has now published their consensus report. This is calling for action to address increasing difficulties in recruiting patients, regulatory complexity, administrative burdens and high costs.

Biocon wins first FDA interchangeable label of Novolog biosimilar

The U.S. FDA on July 15 cleared Biocon Biologics Ltd.’s Kirsty (insulin aspart-xjhz) as the first and only interchangeable biosimilar product referencing Novo Nordisk A/S’ Novolog (insulin aspart), a rapid-acting diabetes medication. Novo won FDA approval of Novolog in June 2000 as an injectable fast-acting human insulin analogue to improve glycemic control in adults and children with type 1 and type 2 diabetes. In the U.S., Kirsty is the second Novolog biosimilar to gain regulatory clearance. Sanofi-Aventis U.S. LLC’s Merilogy (insulin-aspart-szjj) was the first biosimilar to cross the regulatory finish line, without the interchangeable label, in February 2025.

Abivax, Lilly, others to roll out IBD findings with new prospects for UC

Efforts in inflammatory bowel disease continue to occupy drug developers, with data readouts due in the near term. Eagerly awaited are phase III data from Abivax SA with micro-RNA-124-targeting obefazimod in ulcerative colitis. Also among the results ahead are those from Eli Lilly and Co. from the phase IIb study called Emerald-2 with MORF-057 in the same indication. The drug is an oral small-molecule inhibitor of the α4β7 integrin.

June FDA approvals jump, led by three new NMEs

The U.S. FDA approved 23 drugs in June, the highest monthly total so far in 2025 and a jump from 15 approvals in May. Among them were three new molecular entities: CSL Behring LLC’s Andembry (garadacimab) for hereditary angioedema, Merck & Co. Inc.’s Enflonsia (cesrovimab) for respiratory syncytial virus infection and Nuvation Bio Inc.’s Ibtrozi (taletrectinib) for ROS1+ non-small-cell lung cancer.

Also in the news

Ajax, Aldeyra, Amphista, Astrazeneca, Avicanna, Biogen, Blueprint, Bridge Health, Daiichi, Diamedica, Galderma, GC Biopharma, GSK,  Harrow, Harvard, Hyloris, I-Mab, Innovent, Itolerance, J&J, Kadimastem, Kowa, Merck, Molteni Farmaceutici, Nervgen, Nicox, Nkgen, NLS, Novartis, Okyo, Oncozenge, Praxis, Quetzal, Rocket, Samsung Bioepis, Sanofi, Schrödinger, Voyager