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Results For "rare-disease"

234 News Found

Metropolis Healthcare launches Genomics Centre to scale precision diagnostics and research
R&D | January 14, 2026

Metropolis Healthcare launches Genomics Centre to scale precision diagnostics and research

Backed by two CAP - accredited genomics labs in Delhi NCR and Mumbai, supporting oncology, reproductive health, neurology, transplant immunology, and rare diseases


Bristol Myers Squibb reports positive Phase 3 results for Camzyos in adolescents with oHCM
Clinical Trials | January 14, 2026

Bristol Myers Squibb reports positive Phase 3 results for Camzyos in adolescents with oHCM

The study showed a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo


Nxera secures exclusive APAC rights to DMD drug Vamorolone in deal with Santhera
News | January 11, 2026

Nxera secures exclusive APAC rights to DMD drug Vamorolone in deal with Santhera

Nxera will take full responsibility for regulatory approvals, clinical development where required, manufacturing and commercialization in the licensed territories


Italfarmaco and JCR Pharmaceuticals partner to bring Duchenne therapy to Japan
News | December 30, 2025

Italfarmaco and JCR Pharmaceuticals partner to bring Duchenne therapy to Japan

The partnership also sets the stage for broader collaboration on rare disease treatments


SN Bioscience’s SNB-101 wins FDA orphan drug designation for gastric cancer
Clinical Trials | December 29, 2025

SN Bioscience’s SNB-101 wins FDA orphan drug designation for gastric cancer

The drug, currently in a phase 1b/2 clinical trial for small cell lung cancer, is a polymer nanoparticle formulation of SN-38


EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP
Drug Approval | December 27, 2025

EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP

Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation


Ipsen’s FALKON trial falls short in rare FOP study, offers key insights
Clinical Trials | December 22, 2025

Ipsen’s FALKON trial falls short in rare FOP study, offers key insights

FOP is a rare genetic disorder caused by mutations in the ALK2 kinase


Pharma overhaul: EMA hails “Once-in-a-Generation” reform
Policy | December 16, 2025

Pharma overhaul: EMA hails “Once-in-a-Generation” reform

The reform is set to modernise how medicines are developed, authorised, and made available across the EU