Pharmafile Logo
Pharmafocus logo
Advanced Search

Rare Diseases

Showing 15 posts of 33 posts found.

European Medicines Agency recommended 104 medicines for approval in 2025

January 20, 2026
Research and Development European Medicines Agency, Rare Diseases

The European Medicines Agency (EMA) has announced data from 2025 showing that over the course of the year it recommended …

News

Mavatar announces research collaboration with Sobi

January 6, 2026
Research and Development Immunology, Mavatar, Rare Diseases, sobi

Mavatar has announced the initiation of a non-clinical research collaboration with Sobi, using Mavatar’s technology to generate data insights into …

News
640px-eye_iris

NICE recommends treatment for rare genetic eye disorder

August 11, 2025
Medical Communications Chiesi, Leber Hereditary Optic Neuropathy, NHS England, National Institute for Health and Care Excellence, Opthalmology, Rare Diseases

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Cheisi’s idebenone (Raxone) within its …

News

Hansa Biopharma announces positive data for Duchenne muscular dystrophy gene therapy

August 4, 2025
Research and Development Duchenne Muscular Dystophy, Hansa Biopharma, Rare Diseases, Sarepta Therapeutics, clinical trial, genetic disorder

Hansa Biopharma has announced encouraging results from its ongoing SRP-9001-104 trial, which is investigating the use of imlifidase as a …

News
clinical_trial-web

Pharvaris to report phase 3 data and submit New Drug Application

July 15, 2025
Medical Communications, Research and Development Pharvaris, Rare Diseases, US Food and Drug Administration, clinical trial, genetic condition, new drug application

Pharvaris has announced that its data from its phase 3 study of deucrictibant immediate-release capsules (IR) for the treatment of …

News

CSL Behring’s therapy for hereditary angioedema receives positive assessment in Germany

June 26, 2025
Market Access, Market Research, Medical Communications, Research and Development, Sales and Marketing CSL Behring, Institute for Quality and Efficiency in Health Care, Rare Diseases, clinical trials, hereditary angioedema, market access

CSL Behring has announced a key milestone for its hereditary angioedema (HAE) therapy, garadacimab, following a positive benefit assessment by …

News
handshake

Chiesi partners with Key2Brain to develop therapies for rare neurodegenerative diseases

June 12, 2025
Manufacturing and Production, Mergers and Acquisitions, Research and Development Chiesi Group, Key2Brain, Neurology, Rare Diseases, lysosomal storage disorders, neurodegenerative disease, partnership, ultra-rare disease

Italian biopharma, Chiesi Group, and Key2Brain, a Swedish biotech, have agreed to advance the development of two blood-brain barrier (BBB)-crossing …

News

Vertex presents promising new data from cystic fibrosis trials

June 9, 2025
Medical Communications, Research and Development European Cystic Fibrosis Conference, Rare Diseases, Vertex, clinical trial, cystic fibrosis

Vertex Pharmaceuticals has presented new data showing the clinical and quality-of-life benefits of its cystic fibrosis (CF) therapies, particularly Alyftrek …

News
laboratory-testing-az

Trial into rare genetic disease Werner syndrome reports promising results

June 6, 2025
Research and Development Chiba University, Niagen Bioscience, Rare Diseases, Werner Syndrome, clinical trial, rare genetic disease

Researchers at Chiba University, Japan, alongside Niagen Bioscience, have reported successful results from the world’s first trial of nicotinamide riboside …

News
handshake

Sanofi to acquire Blueprint, advancing therapy for rare immunological diseases

June 3, 2025
Mergers and Acquisitions, Research and Development Blueprint Medicines, Immunology, Rare Diseases, Sanofi, advanced systemic mastocytosis (ASM), indolent systemic mastocytosis (ISM), rare immunological diseases

Sanofi and Blueprint Medicines Corporation, a US biopharma, have entered into an agreement under which Sanofi will acquire Blueprint. This …

News

Gene Weaver awarded Gates Foundation grant for programmable gene insertion platform

May 30, 2025
Medical Communications, Research and Development Bill and Melinda Gates Foundation, Gene Weaver, Oncology, Pharmacy, Rare Diseases, funding, gene editing

UK-based biotech Gene Weaver has received $515,000 from the Bill and Melinda Gates Foundation to advance its programmable gene insertion technology …

News

Ochre Bio appoints Elaine Sullivan as senior independent non-executive director

May 30, 2025
Research and Development Infections and infestations, Ochre Bio, Oncology, RNA therapeutics, Rare Diseases, appointment, senior independent non-executive director

Ochre Bio has appointed Elaine Sullivan as senior independent non-executive director. She joins the Oxford-based RNA therapeutics company with a …

Appointment

Hansa Biopharma data demonstrates potential of imlifidase in Guillain-Barré syndrome

May 15, 2025
Research and Development Guillain-Barré Syndrome (GBS), Hansa Biopharma, Immunology, Peripheral Nerve Society (PNS) Annual Meeting, Rare Diseases

Hansa Biopharma will present data from its 15-HMedIdeS-09 phase 2 study of imlifidase for treating Guillain-Barré syndrome (GBS) at the …

News

Kaerus Bioscience secures FDA approval for rare genetic disorder treatment

May 15, 2025
Research and Development FDA, Fragile X syndrome, Kaerus Bioscience, Orphan Drug Designation (ODD), Rare Diseases, Rare Pediatric Drug Designations (RPDD), genetic syndromes

Kaerus Bioscience announces that its lead candidate KER-0193 has been granted both Orphan Drug Designation (ODD) and Rare Pediatric Drug …

News

Ipsen’s Iqirvo approved for use in NHS Scotland to treat rare liver disease

April 8, 2025
Research and Development R&D, Rare Diseases, Scottish Medicines Consortium, ipsen, primary biliary cholangitis (PBC)

The Scottish Medicines Consortium (SMC) has accepted the use of Ipsen’s Iqirvo (elafibranor) in NHS Scotland for the treatment of …

News
The Gateway to Local Adoption Series

Latest content

IsomAb appoints Philip Brainin as CEO

March 19, 2026

Naobios partners with SGS to manufacture RSV challenge agent

March 19, 2026

Qlaris Bio appoints Fred Guerard as President and CEO

March 18, 2026

Under-Secretary of State for Health Innovation and Safety visits new building on University of Huddersfield’s NHIC

March 18, 2026

AEON Biopharma appoints John Bencich as CFO

March 17, 2026