Chiesi inks agreement with AbbVie subsidiary to push next-generation brain-targeting therapies for rare diseases

Need to include ASMD as a notified condition in the National Policy for rare diseases

ASMD is highly variable and the age of onset, specific symptoms and severity of the disorder can vary dramatically from one person to another

Takeda to strengthen health system for rare diseases in India

These initiatives aim to improve access to healthcare for rare disease patients

Sanofi reaffirms commitment to rare diseases in India

Receives Subject Expert Committee recommendation to bring new therapies of Pompe, ASMD to India

Dr Sheela Nampoothiri appointed as co-chair of ICMR's Rare Diseases expert committee

Dr Ratna Puri from Sir Ganga Ram Hospital, Delhi is the Chairperson of the expert committee.

Merck KGaA Q3 2025 net sales remains flat amid geopolitical uncertainty

FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma

Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition

Biohaven disappointed as rare disease therapy faces FDA setback 'despite promising results'

The FDA setback highlights ongoing tensions between patient needs, regulatory processes, and the use of real-world evidence in evaluating treatments for rare diseases

Zydus receives FDA’s ODD for Desidustat for the treatment of beta-thalassemia

Orphan drug designation (ODD) by the USFDA for Desidustat, provides eligibility for a potential seven-year marketing exclusivity subject to the USFDA approval

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours

Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1