The FDA has approved Emcitate as the first treatment for MCT8 deficiency, providing a therapy for the peripheral thyrotoxicosis associated with this ultra-rare genetic disorder.

The FDA approval adds an oral ALK2 inhibitor for patients aged 12 years and older with the ultra-rare genetic disorder fibrodysplasia ossificans progressiva.

The EU authorisation introduces a pharmacological treatment for eligible boys with cerebral adrenoleukodystrophy under exceptional circumstances.

Lexeo Therapeutics has agreed to acquire Mantle Therapeutics for up to $21.3 million, adding four Friedreich ataxia development programmes to its pipeline.

The FDA has approved Aqneursa as the first treatment specifically indicated for ataxia in adults and children with ataxia-telangiectasia weighing at least 15kg.

The FDA has approved Fayuvi as the first gene therapy for children with Sanfilippo syndrome type A, introducing the first treatment designed to address the underlying cause of the rare neurodegenerative disorder.

The FDA has approved Isembyld as the first spinal muscular atrophy treatment designed to directly target muscle loss, for patients aged 2 years and older receiving an SMN2-targeted therapy.

The FDA has approved Zanvastro as the first treatment for Alexander disease in paediatric and adult patients.

The reimbursement decision clears the way for Irish patients with Friedreich’s ataxia to access the first treatment for the rare neurological disease, following prolonged negotiations over cost and clinical value.

The authorisation gives patients aged five years and older the first treatment specifically approved for Rett syndrome across the European Union, with Acadia preparing for an initial German launch in the fourth quarter of 2026.

The designation provides regulatory development incentives for Vanda’s IL-36 receptor inhibitor in the EU and follows orphan status for the investigational therapy in the US and Japan.

The payment strengthens Dimerix's balance sheet while advancing its regional partnering strategy for the Phase III kidney disease therapy DMX-200 across Asia.

The approval provides adults with fibrodysplasia ossificans progressiva with a new treatment option designed to reduce abnormal bone formation and disease flare-ups in an ultra-rare genetic disorder.

The approval introduces the first therapy designed to address the underlying cause of glycogen storage disease type Ia, offering a one-time gene therapy option for eligible patients aged eight years and older.

The acquisition gives BioMarin access to ALE1, a potential first oral treatment for hypophosphatasia, while expanding its rare disease pipeline with a clinical-stage skeletal disease programme.

The acquisition would add a BLA-stage, one-time AAV gene therapy to PTC’s rare disease portfolio, with a rolling US submission expected to complete in the fourth quarter of 2026.

The differing reimbursement positions highlight how health technology assessment decisions can diverge across UK nations, affecting the timing of patient access to new medicines.

The agreement gives Sentynl an exclusive pathway to commercialise the Phase III-ready rare lung disease therapy in the US while providing global manufacturing rights and funding support for late-stage development.

The transaction would give Sobi full ownership of a specialist commercialisation platform spanning seven Latin American markets, building on its $105 million investment in Pint Pharma in 2025.

The 56-week observational findings add longer-term clinical evidence for the eculizumab biosimilar in both complement-inhibitor-naive patients and those previously treated with reference eculizumab.