Regulation

The European Commission has granted orphan drug designation to ABO-101, a CRISPR-based gene editing therapy for primary hyperoxaluria type 1.

The MHRA cleared INmune Bio's paediatric investigation plan in under three months, keeping the company's 2026 UK marketing authorisation application on track.

Elevar's ReFocus202 trial opens enrolment in non-CCA solid tumours, testing lirafugratinib across a broad range of FGFR2-altered cancers globally.

HUTCHMED's FGFR2-targeted oral candidate met its primary endpoint in pretreated intrahepatic cholangiocarcinoma, supporting a China NMPA priority review NDA.

Phathom has fully enrolled 95 adults in its pHalcon-EoE-201 study of vonoprazan, with topline data expected in Q4 2026.

The Paris-based MedTech has combined a €30m Series D2 with a €30m EIB facility to fund US commercialisation of its FDA-cleared polymer repair

Agomab Therapeutics has finalised the design of its NOV-ERA Phase 2b study, with first participants expected to be dosed in the second half of

The Finnish pharma group's oral pan-TEAD inhibitor now holds Orphan Designation on both sides of the Atlantic as its Phase 2 TEADES trial

Japan's health ministry has approved rilzabrutinib for persistent or chronic ITP, backed by Phase 3 data showing a 23% durable platelet response versus zero

Germany's BfArM has cleared Silexion's siRNA candidate SIL204 for a Phase 2/3 trial in locally advanced pancreatic cancer, with first dosing expected

BridgeBio will share primary Phase 3 PROPEL 3 results for oral infigratinib in children with achondroplasia at ICCBH 2026 in Montreal.

Stockholm3 detected 90% of aggressive prostate cancers versus 52% for standard PSA testing in a large population-based screening trial published in Annals of Internal

The European Commission has approved Cenrifki for non-relapsing secondary progressive MS, making it the first therapy to target disability progression in the indication.

USPTO notices of allowance extend INNA-051's potential US exclusivity to at least 2042, covering both composition of matter and rhinovirus-mediated COPD exacerbations.

The Egham-based rare disease pharma company has appointed the former LEO Pharma and Eli Lilly executive to lead its next phase of commercial growth.

Medicenna Therapeutics has added two issued U.S. patents and one allowed application, reinforcing IP protection around bizaxofusp and its broader Superkine pipeline.

Week 52 VELA data show sonelokimab outperforming competing IL-17 agents in hidradenitis suppurativa, with a BLA filing targeted for late September 2026.

Nephros says its 5-nanometre pore filters capture both microplastics and nanoplastics, following the EPA's April 2026 decision to prioritise plastic particle

BioCardia plans to file CardiAMP Cell Therapy for approval in Japan by Q4 2026, targeting an initial patient subgroup of 20,000 ischemic heart failure

Boehringer Ingelheim survival models predict nerandomilast could more than double median IPF survival, based on Phase III FIBRONEER trial data.