Rare Diseases
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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
Biotechnology
Shares of US clinical-stage biotech Vaccinex leapt as much as 233% to an intraday high of $9.56 on Friday, after it disclosed the signing of multi-project deals with two undisclosed prominent pharmaceutical companies. 20 February 2021
Biotechnology
Parisian biotech Advicenne has provided a business update, highlighting a recent positive scientific opinion for lead medicine ADV7103 (potassium citrate/potassium hydrogen carbonate) in Europe. 19 February 2021
Biotechnology
US clinical-stage biopharma company Immunic today announced positive top-line data from an investigator-sponsored Phase II proof-of-concept clinical trial of IMU-838 in primary sclerosing cholangitis (PSC), an uncommon live disease. 18 February 2021
Biotechnology
Oxford University, UK, biotech spinout Evox Therapeutics today announced that it has raised £69.2 million ($95.4 million) in a Series C financing round. 18 February 2021
Biotechnology
The US Food and Drug Administration has approved the supplemental Biologics License Application (sBLA) for Panzyga (immune globulin intravenous [Human] – ifas 10% liquid preparation) to treat adult patients with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP). 15 February 2021
Biotechnology
Takeda Pharmaceutical has announced new, late-breaking Phase III data from the TAK-620-303 (SOLSTICE) trial, for the investigational drug TAK-620 (maribavir) which met its primary endpoint of superiority compared to conventional antiviral therapies. 15 February 2021
Biotechnology
AbbVie has picked up a twelfth indication for Botox (onabotulinumtoxinA), opening a new revenue stream in the therapeutic and cosmetic workhorse’s sunset years. 10 February 2021
Pharmaceutical
Shares of USA and UK-based KalVista Pharmaceuticals went stratospheric, after the clinical-stage pharma company announced positive top-line Phase II clinical trial results demonstrating statistically- and clinically-significant efficacy of KVD900 as an oral on-demand treatment for hereditary angioedema (HAE) attacks, a rare inherited disorder. 9 February 2021
Pharmaceutical
Bristol Myers Squibb has won approval in Europe for Inrebic (fedratinib), to treat enlarged spleen in certain people with myelofibrosis, a rare bone marrow disorder. 9 February 2021
Pharmaceutical
The Russian government has officially approved a procedure for the purchase of some high-priced drugs for children with orphan diseases, according to recent statements by the press-service of the Russian government. 9 February 2021
Biotechnology
The Russian Ministry of Health has begun centralized procurements of Ocrevus (ocrelizumab) - a drug for the treatment of the most severe form of multiple sclerosis, reports The Pharma Letter’s local correspondent. 8 February 2021
Biotechnology
On Friday, the US Food and Drug Administration granted accelerated approval for Ukoniq (umbralisib), a kinase inhibitor including PI3K-delta and casein kinase CK1-epsilon. 8 February 2021
Pharmaceutical
Japanese drugmaker Takeda has reported revenue for its first three quarters of the financial year that slipped below the previous year’s levels, but profits that soared. 4 February 2021
Biotechnology
Ireland-headquartered biopharma Horizon Therapeutics is to acquire US biotech Viela Bio for $3.05 billion. 1 February 2021
Biotechnology
Hypereosinophilic syndrome (HES) refers to a group of rare blood disorders that are characterized by a marked overproduction of eosinophils (white blood cells that lay an important role in immunity). While the exact prevalence of HES is unknown, the World Health Organization reported age-adjusted incidence of about 3-4 people per 10,000,000 between 2001 and 2005. 28 January 2021
Pharmaceutical
Agios Pharmaceuticals has announced positive results from the Phase III ACTIVATE trial of mitapivat. 28 January 2021
Pharmaceutical
Recordati has announced that the US Food and Drug Administration (FDA) has approved a new indication for Carbaglu (carglumic acid) tablets 200mg. 27 January 2021
Biotechnology
Boston, USA-based Albireo Pharma - a spinoff of UK-based AstraZeneca - is moving ahead with the regulatory process for odevixibat. 25 January 2021
Biotechnology
Shares of Swedish Orphan Biovitrum, also known as Sobi, gained more than 2% in early trading, as the company revealed that the European Commission has approved an extension of the indication for Doptelet (avatrombopag) to include the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (eg, corticosteroids, immunoglobulins) in all European Union member states. 20 January 2021
Pharmaceutical
Anglo-Irish rare diseases specialist Amryt Pharma (LSE: AMYT) has won a positive reimbursement decision in the UK for Myalepta (metreleptin). 20 January 2021
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