Oral semaglutide gets new FDA approval
The FDA has approved a new indication for Novo Nordisk’s oral semaglutide Rybelsus. The drug is now indicated for reducing the risk of major adverse cardiovascular events, including cardiovascular death, heart attack, and stroke in at-risk adults with type II diabetes. In the SOUL clinical trial, oral semaglutide 14 mg reduce the risk of major adverse cardiovascular events by 14 percent over four years compared to placebo.
Rybelsus was initially approved in 2019.
Promising phase 1 mAb malaria study
A monoclonal antibody developed by researchers at the University of Maryland School of Medicine’s Center for Vaccine Development and Global Health (CVD) has shown promise in preventing malaria in a phase I trial. The antibody, named MAM01, is designed to provide passive immunity by targeting a protein on the outer surface of the parasite Plasmodium falciparum, which is responsible for most severe malaria cases. The goal is to block infection before it reaches the bloodstream.
Healthy volunteers received the drug and exposed to bites from mosquitoes infected with the malaria parasite in a controlled challenge trial. None of the volunteers who received the highest dose developed an infection.
“The CVD research team is now exploring optimized dosing and cost-reduction strategies to make monoclonal antibody-based prevention feasible in malaria-endemic regions,” said School of Medicine Dean Mark T. Gladwin. “Testing of this preventive treatment has already started in young children in Uganda, based on the promising results from the first trial conducted here.”
Rights to Rett Syndrome gene therapy returned to Taysha
Taysha Gene Therapies has regained full rights to the lead compound TSHA-102 program for Rett syndrome after a deadline expired for collaborator Astellas to exercise its option for transaction negotiations. In accordance with an agreement made in 2022, Taysha delivered a data package to Astellas that included data from the Part A REVEAl clinical trial.
“We greatly appreciate the collaborative relationship we’ve had with Astellas, and the progress made to date across our TSHA-102 program, including recent clinical data further highlighting the therapy’s broad and consistent impact and the receipt of Breakthrough Therapy designation from the FDA,” said Sean P. Nolan, Chairman and CEO of Taysha. “We are thrilled to regain full global rights to our lead TSHA-102 Rett syndrome program, which enables full strategic flexibility and optionality as we continue to advance the program and focus on driving long-term value.”
A collaboration for pandemic preparedness
The Coalition for Epidemic Preparedness Innovations (CEPI) and the Serum Institute of India (SII) have launched a collaboration to enhance global pandemic preparedness by developing vaccines targeting H5N1 avian influenza.
Supported by up to $16.4 million in CEPI funding, the initiative will use a baculovirus-based platform to produce and compare two H5 antigens for a recombinant protein vaccine: a wild-type and an AI-optimized antigen. The project will also test how rapidly the baculovirus system can generate antigens during a simulated outbreak and serve as a proof of concept for AI-assisted vaccine design.
Richard Hatchett, CEO of CEPI, said: “With a potential pandemic influenza vaccine candidate already in development on a validated platform, and with a vaccine manufacturing juggernaut ready to go, the world’s disease defenses will be poised to respond swiftly with new vaccines, potentially in 100 days, should a flu virus erupt into a potentially deadly and fast-spreading human pandemic.”
Pfizer medicine to be suspended from EU market
EMA’s CHMP has recommended stripping Pfizer’s Oxbryta of its marketing authorization. The medicine has been suspended since September 2024 when Pfizer voluntarily withdrew it from all markets while a safety review was carried out following a higher than expected number of deaths. The CHMP has now concluded that the benefits of the medicine no longer outweigh the risks. Recent clinical trials have shown that the medicine can be associated with more sudden pain episodes and deaths, although the reasons for this are unknown.
“The CHMP found no clear explanation for the increased risks and could not identify measures to effectively minimise these risks or any subgroup of patients for whom the medicine’s benefits would outweigh its risks,” said a statement from the EMA. “As a result, the CHMP concluded that the benefit-risk balance of Oxbryta is no longer favourable, and the suspension of the medicine’s marketing authorisation should remain in place.”
Speciality pharmacy launches in Ohio
Orsini Specialty Pharmacy has opened a new facility in Columbus, Ohio, integrating specialty pharmacy services with third-party logistics operations. The site will provide distribution, dispensing, and logistics support for complex and rare disease therapies. The integrated model is intended to streamline access, supply chain efficiency, and patient support through a unified infrastructure. Orsini stated that the facility strengthens its national service capabilities and supports its existing partnerships with manufacturers across specialty and cell and gene therapy markets.
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