Kedrion SpA’s investigational plasma-derived treatment for congenital aceruloplasminemia has been awarded European orphan drug designation by the EMA. The company is working to advance this treatment toward clinical development in Europe.

Fifteen years since the first patient was treated, and after being ditched by two companies, the EMA is recommending approval of Waskyra (etuvetidigene autotemcel), the first gene therapy for treating Wiskott-Aldrich syndrome.

Proqr Therapeutics NV has received clinical trial application (CTA) authorization under the EMA’s new centralized review process for a phase I study of AX-0810, which is being developed for the treatment of cholestatic diseases such as primary sclerosing cholangitis and biliary atresia.

There was a curate’s egg for Sanofi SA from this month’s meeting of the EMA’s Committee for Medicinal Products for Human Use, with a recommendation to approve one of the French pharma’s drugs – and the rejection of another.

Taho Pharmaceuticals Ltd. announced an NDA submission to the U.S. FDA for TAH-3311, developed as the world’s first oral dissolving film formulation of apixaban, marking a regulatory milestone for the Taipei-based biotech.

Taho Pharmaceuticals Ltd. announced an NDA submission to the U.S. FDA for TAH-3311, developed as the world’s first oral dissolving film formulation of apixaban, marking a regulatory milestone for the Taipei-based biotech.

The U.S. FDA approved Merck & Co. Inc.’s Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) injection on Sept. 19, making it the first and only subcutaneously (SC)-administered immune checkpoint inhibitor that can be administered in about a minute.

The U.S. FDA approved Merck & Co. Inc.’s Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) injection on Sept. 19, making it the first and only subcutaneously (SC)-administered immune checkpoint inhibitor that can be administered in about a minute.

Following the first approvals in the U.K., Canada, Australia and Switzerland, the EMA’s Committee for Medicinal Products for Human Use (CHMP) is now recommending approval of Bayer AG’s Lynkuet (elinzanetant), a non-hormonal treatment for symptoms of menopause. The drug, the first dual neurokinin-1 and neurokinin-3 receptor antagonist, is for the treatment of moderate to severe vasomotor symptoms, also known as hot flashes.

The European Commission (EC) posted a call for evidence regarding its intent to “simplify EU rules” for medical technology with a comment period that began Sept. 8, 2025. Stakeholders almost immediately flooded the docket with recommendations.