Regulatory Science

Rafael Holdings hit last-patient-last-visit in its pivotal TransportNPC trial and expects to file an NDA for Trappsol Cyclo in the second half

Palopegteriparatide sustained multi-organ benefits over five years in hypoparathyroidism, with 82% of patients meeting the multi-component response endpoint.

ESLIM-02 Phase III data showed a 66% durable response rate versus 15% for placebo, supporting a priority-review NDA filed with China's NMPA.

Humacyte's bioengineered vessel delivered 91 more catheter-free days than standard-of-care AV fistula in female dialysis patients, triggering a supplemental BLA filing.

The Canadian GMP manufacturer completed its first UK psilocybin export, supplying 5mg capsules and biomass under Health Canada and UK Home Office authorisations.

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

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

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

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

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.

Agios will pay $25m upfront for global rights to cevidoplenib, a next-generation oral SYK inhibitor targeting immune thrombocytopenia, with Phase 3 planned for 2028.

Alvotech has resubmitted US licence applications for AVT05 and AVT06 following FDA manufacturing queries at its Reykjavik facility, with a six-month review expected.

PharmAla will receive up to $100m in milestones and royalties for exclusive U.S. rights to its non-racemic MDMA candidate ALA-002.

The Dijon-based emergency-medicines specialist issued new shares and warrants to fund regulatory steps for its needle-free seizure treatment and ramp production capacity.

The Providence-based medtech has cleared FDA's IDE process, unlocking a 120-subject pivotal RCT comparing Separo to standard mucosal cautery vasectomy.

The European Commission has expanded lomitapide's indication to children aged five and older with homozygous familial hypercholesterolaemia, backed by Phase 3 data.