Rare Diseases
European Medicines Agency recommended 104 medicines for approval in 2025
January 20, 2026Research 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 …
Mavatar announces research collaboration with Sobi
January 6, 2026Research 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 …
NICE recommends treatment for rare genetic eye disorder
August 11, 2025Medical 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The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Cheisi’s idebenone (Raxone) within its …
Hansa Biopharma announces positive data for Duchenne muscular dystrophy gene therapy
August 4, 2025Research 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 …
Pharvaris to report phase 3 data and submit New Drug Application
July 15, 2025Medical 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 …
CSL Behring’s therapy for hereditary angioedema receives positive assessment in Germany
June 26, 2025Market 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 …
Chiesi partners with Key2Brain to develop therapies for rare neurodegenerative diseases
June 12, 2025Manufacturing 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 …
Vertex presents promising new data from cystic fibrosis trials
June 9, 2025Medical 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 …
Trial into rare genetic disease Werner syndrome reports promising results
June 6, 2025Research 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 …
Sanofi to acquire Blueprint, advancing therapy for rare immunological diseases
June 3, 2025Mergers 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 …
Gene Weaver awarded Gates Foundation grant for programmable gene insertion platform
May 30, 2025Medical 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 …
Ochre Bio appoints Elaine Sullivan as senior independent non-executive director
May 30, 2025Research 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 …
Hansa Biopharma data demonstrates potential of imlifidase in Guillain-Barré syndrome
May 15, 2025Research 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 …
Kaerus Bioscience secures FDA approval for rare genetic disorder treatment
May 15, 2025Research 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 …
Ipsen’s Iqirvo approved for use in NHS Scotland to treat rare liver disease
April 8, 2025Research 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 …
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