Latest Rare Diseases News

Zelira Therapeutics has raised nearly US$33 million from ThirdGate Capital to advance its HOPE® 1 drug targeting autism-related Phelan-McDermid Syndrome through FDA clinical trials. This funding milestone sets the stage for pivotal regulatory progress in the US.

Neuren Pharmaceuticals’ partner Acadia projects DAYBUE sales to reach around US$700 million by 2028, driven by US market expansion and international approvals. The launch of a new formulation and ongoing clinical trials in Japan underpin this optimistic outlook.

Neuren Pharmaceuticals has published promising Phase 2 trial results for NNZ-2591 in Phelan-McDermid syndrome, underpinning its ongoing Phase 3 Koala trial recruiting in the US.

Neuren Pharmaceuticals has secured Health Canada approval to expand its pivotal Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome into Canadian sites, complementing ongoing US enrolment.

BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics for US$4.8 billion includes Dimerix’s US licensing agreement, promising significant milestone payments and royalties. This deal strengthens Dimerix’s position as it advances its Phase 3 kidney disease therapy.

Opthea Limited has announced a strategic pivot to develop OPT-302 for Lymphangioleiomyomatosis (LAM), a rare lung disease affecting women, aiming for ASX reinstatement in 2026. The company leverages strong intellectual property and cash reserves to pursue orphan drug designation and clinical development.

Chimeric Therapeutics has won FDA Orphan Drug Designation for its CHM CDH17 CAR-T therapy targeting gastric cancer, advancing its clinical development with regulatory incentives and potential market exclusivity.

Neuren Pharmaceuticals reported a milestone quarter with DAYBUE net sales reaching US$101.1 million, driving a 24% jump in royalty income. The expanding patient base and community physician uptake signal strong growth ahead.

Pathkey.AI reports strong validation of its TrialKey AI platform’s predictive accuracy and announces key leadership changes as it advances toward commercial scale.

Neurotech International has initiated an Authorised Prescriber program for its lead therapy NTI164, expanding access for children with neurodevelopmental disorders in Australia. The company also secured a Rare Pediatric Disease Designation from the US FDA for NTI164 in Rett Syndrome, marking a key regulatory milestone.

PYC Therapeutics has kicked off a global Phase 1b repeat dose study of its investigational drug PYC-001 targeting Autosomal Dominant Optic Atrophy, a rare genetic eye disease with no current treatments. Initial dosing has begun, with key data expected in the second half of 2026.

Neuren Pharmaceuticals has secured FDA Fast Track designation for its drug NNZ-2591 targeting Phelan-McDermid syndrome, accelerating its path through clinical development. This milestone underscores the urgent need for treatments in rare neurodevelopmental disorders.