Rare Diseases
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Biotechnology
Wave Life Sciences has announced positive interim data from the ongoing Phase II FORWARD-53 study of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. 25 September 2024
Pharmaceutical
Japanese drugmaker Eisai closed down 2.3% at 5,587 yen today, despite news of regulatory approvals for two of its drugs. 24 September 2024
Biotechnology
Aptadir Therapeutics, a biotech company focused on RNA inhibitor-based therapies for cancer and genetic conditions, has launched with $1.6 million in pre-seed funding. 24 September 2024
Pharmaceutical
The European Commission has conditionally approved the marketing application submitted by Ipsen for Iqirvo (elafibranor) 80mg tablets for the treatment of primary biliary cholangitis (PBC) 21 September 2024
Pharmaceutical
Today, the US Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). 20 September 2024
Pharmaceutical
Applied Therapeutics saw its stock price climb by over two-thirds after an encouraging update on the US Food and Drug Administration review of govorestat. 19 September 2024
Biotechnology
TG Therapeutics yesterday shared new five-year data from the ULTIMATE I & II Phase III trials evaluating Briumvi (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS). 19 September 2024
Biotechnology
Canada’s NanoVation Therapeutics, a privately-held platform company developing innovative technologies to overcome the barriers of nucleic acid delivery, has announced a multi-year partnership with Danish drugmaker Novo Nordisk. 18 September 2024
Biotechnology
Ascendis Pharma has announced positive topline data from the pivotal Phase II ApproaCH trial of TransCon CNP (navepegritide). 18 September 2024
Biotechnology
AstraZeneca’s Fasenra (benralizumab) has been approved in the USA for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA, the UK pharma major announced today. 18 September 2024
Biotechnology
US biotech Capricor Therapeutics has entered into a binding term sheet with Japanese drugmaker Nippon Shinyaku for the commercialization and distribution in Europe of Capricor’s lead asset, deramiocel. 17 September 2024
Pharmaceutical
Dystonia is a neurological movement disorder characterized by involuntary muscle contractions that cause repetitive movements, abnormal postures, or twisting. 17 September 2024
Biotechnology
The US Food and Drug Administration (FDA) has approved Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS), from Swiss pharma giant Roche. 16 September 2024
Pharmaceutical
The Gastrointestinal Drugs Advisory Committee (GIDAC) of the US Food and Drug Administration (FDA) met on Friday to discuss Intercept Pharmaceuticals’ supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA). 16 September 2024
Pharmaceutical
Boehringer Ingelheim today announced that the FIBRONEER-IPF trial met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] (FVC) at week 52 versus placebo. FVC is a measure of lung function. 16 September 2024
Pharmaceutical
Oligonucleotide therapeutics are rapidly emerging as a promising class of drugs capable of addressing a variety of genetic and complex diseases by targeting RNA sequences.1 16 September 2024
Pharmaceutical
Thursday was a wretched day for Fulcrum Therapeutics, a US biopharma company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases. 13 September 2024
Biotechnology
California, USA-based biotech BridgeBio Pharma saw its shares dip 4% to $29.60 yesterday on disappointing clinical results and the company’s decision to drop an early-stage candidate. 11 September 2024
Biotechnology
Ionis Pharmaceuticals has announced plans to raise $500 million through a public offering. 11 September 2024
Pharmaceutical
Santhera Pharmaceuticals has signed an exclusive distribution agreement with GENESIS Pharma for Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in 20 markets in Central and Eastern Europe. 10 September 2024
Biotechnology
Privately-held Swiss biotech AB2 Bio has entered into an option and licensing agreement with Japan’s Nippon Shinyak, which it describes as having extensive experience in marketing rare disease therapeutics in the USA. 27 January 2025
Biotechnology
Swiss pharma giant Roche today announced positive top-line results from year two of the EMBARK trial, a global, randomized, double-blind Phase III study of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy. 27 January 2025
Pharmaceutical
Neurocrine Biosciences has announced the appointment of Andrew Ratz as senior VP of drug development, supporting the firm’s expanding R&D efforts. 24 January 2025
Pharmaceutical
US clinical-stage drug developer Stealth BioTherapeutics announced that the US Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) action date for the New Drug Application (NDA) for elamipretide, a first-in-class mitochondria-targeted therapeutic in development for individuals with Barth syndrome. 24 January 2025
Biotechnology
The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is being developed for the treatment of Duchenne muscular dystrophy (DMD) by Japanese drugmaker Nippon Shinyaku. 24 January 2025
Biotechnology
USA-based healthcare giant Johnson & Johnson has announced the publication of key Phase III data for nipocalimab in The Lancet Neurology. 23 January 2025
Biotechnology
The US Food and Drug Administration (FDA) has accepted Biogen’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen, marketed as Spinraza, for spinal muscular atrophy (SMA). 23 January 2025
Biotechnology
USA-based biotech IMMvention Therapeutix yesterday revealed it has entered into a strategic collaboration and license agreement with Novo Nordisk to co-develop oral therapies for sickle cell disease (SCD) and other chronic conditions. 23 January 2025
Biotechnology
US genetic meds company MeiraGTx Holdings yesterday revealed that the US Food and Drug Administration (FDA) has granted the company Rare Pediatric Disease (RPD) designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1). 23 January 2025
Pharmaceutical
The US Food and Drug Administration (FDA) is warning about the risk of a rare but serious allergic reaction with the medicine glatiramer acetate (marketed under trade names including Copaxone, Glatopa), which is used to treat patients with multiple sclerosis (MS). 22 January 2025
Biotechnology
Kyverna Therapeutics (Nasdaq: KYTX), a California biotech developing CAR-T therapies for autoimmune diseases, has appointed Naji Gehchan as chief medical officer. 22 January 2025
Pharmaceutical
Rett syndrome is a rare neurological and developmental disorder that leads to a gradual decline in motor skills and language. An X-linked genetic disease, Rett syndrome occurs in approximately 1 in every 10,000 female births each year.1 22 January 2025
Biotechnology
Dutch biotech Pharming Group today announced that its board of directors has nominated Fabrice Chouraqui to become the company’s new executive director and chief executive, succeeding Sijmen de Vries. 21 January 2025
Pharmaceutical
French pharmaceutical company Servier and Google Cloud have announced an expanded five-year partnership aimed at leveraging artificial intelligence (AI) and generative AI to accelerate drug research and development. 16 January 2025
Pharmaceutical
The UK’s health technology assessor, the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance that recommends Agamree (vamorolone) for use in the National Health Service (NHS) in England, Wales and Northern Ireland for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older. 16 January 2025
Biotechnology
Shares of US biotech Regenxbio surged 18.4% in pre-open trading Tuesday after announced a strategic partnership with Japan’s Nippon Shinyaku. 15 January 2025
Biosimilars
South Korean biosimilars developer Samsung Bioepis and Israel’s Teva Pharmaceutical Industries have entered into a license, development and commercialization agreement for Epysqli (eculizumab-aagh), Samsung Bioepis’ copy of Soliris (eculizumab), in the USA. 11 January 2025
Biotechnology
Maryland, USA-based Veralox Therapeutics has picked up an option to acquire Nudge Therapeutics, along with its portfolio of preclinical cGAS inhibitor compounds. 10 January 2025
Biotechnology
Intellia Therapeutics, a US gene editing company developing CRISPR-based therapies, announced late Thursday its strategic priorities and key anticipated 2025 milestones. 10 January 2025
Biosimilars
The US Food and Drug Administration approved 50 novel drugs in 2024, continuing to outpace its global regulatory peers. 10 January 2025
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