Regulation

Rafael Holdings has reached last-patient-last-visit in its 94-patient TransportNPC trial, with topline data and an NDA submission both expected in H2

The Watertown biotech enrolled 191 patients across 14 countries; topline results for the PH-HFpEF candidate are expected in early Q1 2027.

Teva paid $700 million upfront for Emalex Biosciences, acquiring a Phase 3-complete D1 antagonist with an NDA filing expected in the second half of

The New Jersey biotech's once-nightly, low-sodium oxybate candidate secured ODD based on its potential to eliminate the disruptive mid-night second dose.

Alterity's End-of-Phase 2 meeting secured FDA agreement on trial design, dosing, and endpoints for its pivotal ATH434 programme in multiple system atrophy.

The Cambridge biotech can now advance directly into a randomised Phase 1/2 study of its oral AKT1 inhibitor in hereditary haemorrhagic telangiectasia.

The Paris-based biotech secured RMAT status for its CD22-targeting off-the-shelf CAR-T candidate in relapsed or refractory B-cell acute lymphoblastic

Health Canada has accepted Chiesi's birch triterpenes gel for priority review, potentially making it the first therapy approved for EB wounds in Canada.

Disc Medicine said a Type A FDA meeting confirmed the Phase 3 APOLLO study could support a traditional approval for bitopertin in EPP, with data

CTA313 targets CD19 and BCMA in a Phase 1b basket trial spanning lupus, progressive MS, and autoimmune encephalitis.

An independent data monitoring committee found the study unlikely to demonstrate sufficient efficacy; no safety concerns were identified with riliprubart.

Subcutaneous BRIUMVI hit MCID in 82% of myasthenia gravis patients at Week 24, prompting a potentially registration-directed Phase 2 sequential-therapy trial.

The Cambridge biotech has initiated its pivotal NORTHSTAR study of oral gildeuretinol in Stargardt disease, with no approved treatment currently available.

The Iceland-based biosimilar specialist said the FDA has accepted its BLA for AVT16, a proposed interchangeable biosimilar to Takeda's Entyvio, for ulcerative colitis

Artiva Biotherapeutics secured RMAT designation for its allogeneic NK cell therapy after Phase 2a data showed durable responses in refractory rheumatoid arthritis.

New VITESSE subgroup analysis shows VIASKIN Peanut Patch hit significance across asthma, food allergy and atopic dermatitis co-morbidities in children.

All 12 patients in a UChicago Medicine pilot achieved insulin independence, with mean HbA1c of 5.4% and no severe hypoglycaemic episodes post-transplant.

A 307-patient pivotal trial showed twiist reduced HbA1c by 0.7 points and raised time in range by 16 percentage points over 13 weeks.

RemeGen's telitacicept becomes the first approved therapy for Sjögren's disease in China, backed by Phase 3 data showing significant improvements on both ESSDAI

China's NMPA has conditionally approved telitacicept for IgAN, marking the fourth indication for the dual BAFF/APRIL inhibitor and its first kidney disease clearance.