Rare Diseases
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Biotechnology
At its February meeting, the European Medicines Agency’s human medicines committee CHMP recommended approval of eight novel medicines, one of which is a biosimilar. 24 February 2023
Biotechnology
Shares of Nektar Therapeutics sank more than 40% to $1.78 in pre-market activity today after the company reported that a study of rezpegaldesleukin (rezpeg) did not achieve its goals and that partner Eli Lilly has decided not to proceed with Phase III development of the investigational treatment for systemic lupus erythematosus (SLE). 24 February 2023
Biotechnology
Rare Disease Day is honored globally on February 28 each year. For a long time, the number of known rare diseases in the world was pegged between 6,000 and 8,000. 23 February 2023
Biotechnology
Netherland-based biotech Azafaros today announced its progress in ongoing interactions with health authorities regarding its lead asset, AZ-3102. 20 February 2023
Pharmaceutical
Long approved in Europe, family-owned Italian drugmaker Chiesi Farmaceutici has now received marketing authorization for its Lamzede (velmanase alfa-tycv) in the USA. 17 February 2023
Generics
Patients in India suffering from cystic fibrosis are petitioning the Indian government to allow generic versions of Vertex Pharmaceuticals’ (Nasdaq: VRTX) Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) and Orkambi (lumacaftor/ivacaftor). 17 February 2023
Biotechnology
US biotech Capricor Therapeutics has entered into a partnership with mid-sized Japanese drugmaker Nippon Shinyaku, for the exclusive commercialization and distribution in Japan of Capricor’s lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD), a rare neuromuscular disease with limited treatment options. 16 February 2023
Biotechnology
Dutch rare disease specialist Pharming has suffered a setback in the European approval process for its leniolisib, a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. 16 February 2023
Biotechnology
February 15, 2023
US biotech major Biogen today reported financials for 2022, showing that fourth quarter revenues were $2,544 million, a decline of 7% but beating consensus forecasts of $2.45 billion. 15 February 2023
Biotechnology
Last week’s US Food and Drug Administration (FDA) clinical hold on genetic medicines company 4D Molecular Therapeutics Fabry disease (FD) gene therapy program (4D-310) could benefit fellow US drugmaker Sangamo Therapeutics. 14 February 2023
Biotechnology
French drugmaker Sanofi has announced the availability of its new Pompe disease drug Nexviadyme (avalglucosidase alfa) on the UK’s National Health Service (NHS). 8 February 2023
Biotechnology
The European Commission (EC) marketing authorization (MA) that was granted in December to US biotech Atara Biotherapies for Ebvallo (tabelecleucel) has been transferred to privately French drugmaker Pierre Fabre. 8 February 2023
Pharmaceutical
Relief Therapeutics has decided to voluntarily withdraw its planned initial public offering (IPO), the Swiss biopharma company announced today, when its shares dipped 1%. 8 February 2023
Biotechnology
US biopharma Vertex Pharmaceuticals has reported its fourth-quarter and full-year financial results. 8 February 2023
Biotechnology
When Johnson & Johnson’s Janssen unit acquired nipocalimab in its $6.5 billion buy of Momenta Pharmaceuticals in 2020, the opportunity in myasthenia gravis appeared most compelling.
But the importance of another indication is becoming clear after Janssen’s presentation of positive Phase II results from the UNITY trial for the treatment of pregnant adults at high risk for severe hemolytic disease of the fetus and newborn (HDFN). 7 February 2023
Biotechnology
Roche’s (ROG: SIX) ambition to muscle in on the paroxysmal nocturnal hemoglobinuria (PNH) sector has received another boost, as the Swiss pharma giant today announced positive results from the global Phase III COMMODORE 2 study, evaluating the efficacy and safety of crovalimab in people with PNH who have not been previously treated with complement inhibitors. 7 February 2023
Biotechnology
The US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) to expand the use of Takhzyro (lanadelumab-flyo) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients aged two to 6 February 2023
Biotechnology
In order to spur research and innovation in the pharmaceutical industry, the Narendra Modi government in India is developing a new policy that will cater to a range of therapies - from gene therapy to precision medicine. 6 February 2023
Biotechnology
Swedish Orphan Biovitrum, also known as Sobi, today announced an executive appointment. 2 February 2023
Pharmaceutical
Roche’s shares dipped 1.4% to 322.60 francs this morning, when the Swiss pharma giant announced that, in full-year 2022, it achieved sales growth of 2% (+1% in Swiss francs) to 63.3 billion francs ($.66.8 billion), but warned that 2023 profits would see a downturn. 2 February 2023
Pharmaceutical
Swiss pharma giant Novartis today announced an upgrade to its mid-term guidance, in advance of its Meet Novartis Management event for investors and analysts in London. 21 November 2024
Biotechnology
US biotech major Amgen has announced that Dr Howard Chang will join the company as senior vice president of research, effective December 16, 2024. 21 November 2024
Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Cellular immunotherapy specialist Fate Therapeutics soared as much as 73%, and closed up 12.45 at $2.27 yesterday, after it released early-stage data on its FT819 in lupus. 19 November 2024
Pharmaceutical
US antiviral giant Gilead Sciences on Friday unveiled two-and-a-half-year interim analysis from the ongoing Phase III ASSURE study of its Livdelzi (seladelpar). 16 November 2024
Pharmaceutical
French drugmaker Ipsen announced late-breaking data for Iqirvo (elafibranor 80mg tablets) from an interim analysis of the ongoing open-label extension of the Phase III ELATIVE study at the American Association for the Study of Liver Disease (AASLD) congress. 16 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Biotechnology
Dutch firm Argenx and China’s Zai Lab have received an approval from the National Medical Products Administration (NMPA) for Vyvgart Hytrulo (efgartigimod alfa). 13 November 2024
Pharmaceutical
AstraZeneca and Merck & Co announced that their jointly developed drug Koselugo (selumetinib) has shown positive outcomes in the KOMET Phase III trial. 12 November 2024
Biotechnology
Geron Corporation, a biopharma company focused on telomerase inhibition therapies, has announced a $375 million financing agreement aimed at supporting the commercial rollout of Rytelo (imetelstat). 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Pharmaceutical
Research results featured heavily in the news last week, first with Viking Therapeutics releasing promising early-stage data for its investigational medicine VK2738 at the ObesityWeek meeting. US biotech Beam Therapeutics released early-stage data on its sickle cell disease candidate BEAM-101. Also of note, Arcus Biosciences and Gilead Sciences announced promising Phase III results for their TIGIT inhibitor domvanalimab in combination with zimberelimab. Denmark’s Novo Nordisk released financial results that attracted interest. 10 November 2024
Pharmaceutical
Italian pharma major Recordati saw its shares dip 3.75% to 50.10 euros this morning, despite posting a set of strong financial results. 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Pharmaceutical
US drugmaker Sarepta Therapeutic saw its shares gain 2.4% to $127.97 as it reported financial results for the third quarter 2024. 7 November 2024
Pharmaceutical
USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024
Biotechnology
UK health technology assessor the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Ayvakyt (avapritinib) from USA-based Blueprint Medicines. 6 November 2024
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