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Biotechnology
Last week’s regulatory news included a US Food and Drug advisory committee vote equally for and against approval of ChemoCentryx’ vasculitis treatment candidate avacopan. Also, US vaccine developer Novavax last Monday announced that it is delaying the regulatory filing for its COVID-19 candidate NVX-CoV2373. Research news saw US biotech Curis presenting early-stage data for its CA-4948 for myeloid leukemia and myelodysplastic syndrome, which clearly pleased investors. On the negative side, Biogen released Phase II/III results for its cotoretigene toliparvovec (BIIB112) in a retinal disease that failed to meet its primary endpoint. 16 May 2021
Pharmaceutical
German family-owned pharma major Boehringer Ingelheim has provided important new evidence about the longer-term use of Ofev (nintedanib), which was approved in the USA in September 2019 and in Europe in March 2020 for use in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), a rare autoimmune disease. 14 May 2021
Pharmaceutical
US-headquartered Strongbridge Biopharma saw its shares jump as much as 6% this morning, after the rare diseases specialist revealed that the US Food and Drug Administration has accepted for review the company’s New Drug Application (NDA) for Recorlev (levoketoconazole) for the treatment of endogenous Cushing’s syndrome. 13 May 2021
Pharmaceutical
UK endocrine diseases specialist Diurnal Group has extended its exclusive license agreement with the specialty pharma company, Citrine Medicine, to include the registration, distribution and marketing of Efmody (hydrocortisone modified-release hard capsules – previously named Chronocort) covering China, Hong Kong, Taiwan and Macau. 12 May 2021
Pharmaceutical
Japan’s largest drugmaker Takeda Pharmaceutical today announced financial results for fiscal year 2020 (period ended March 31, 2021), which the company says demonstrate its “resilient portfolio.” 11 May 2021
Pharmaceutical
Japanese pharma company Sumitomo Dainippon Pharma is to acquire the exclusive development and marketing rights in China, Hong Kong, Taiwan and other Asian markets for multiple development compounds from Sinovant, a subsidiary of Roivant Sciences. 10 May 2021
Biotechnology
An evenly split vote from the US regulator’s scientific panel sent shares in California’s ChemoCentryx into free fall on Friday. 10 May 2021
Biotechnology
A 23,650% first quarter revenue increase makes clear the level of fiscal impact a successful coronavirus vaccine can have. 6 May 2021
Biotechnology
Irish rare diseases firm Amryt Pharma has signed a definitive agreement to acquire US-headquartered Chiasma in an all-stock combination valued at around $330 million. 6 May 2021
Biotechnology
Shares in US gene therapy company Avrobio closed 19% lower on Monday. 4 May 2021
Biotechnology
Shares of Sarepta Therapeutics closed up nearly 9% at $77.11 yesterday, after the US rare diseases specialist announced positive results from Part A of the MOMENTUM study (Study 5051-201), a global, Phase II, multi-ascending dose clinical trial of SRP-5051, its next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. 4 May 2021
Biotechnology
The European Commission (EC) has granted marketing authorization to ADV7103 (potassium citrate/potassium hydrogen carbonate), proposed trade name Sibnayal, for the treatment of distal renal tubular acidosis (dRTA), says French biotech Advicenne. 4 May 2021
Pharmaceutical
Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici, announced that the US Food and Drug Administration has approved Ferriprox (deferiprone) for the treatment of patients with transfusional iron overload due to sickle cell disease (or other anemias) in adults and children (age three and older). 4 May 2021
Biotechnology
Notable news last week included AstraZeneca and Sanofi presenting new Phase III date on their jointly-developed nirsevimab in respiratory syncytial virus (RSV). With a heavy week of first-quarter 2021 financial result reporting, attention focussed on Bristol Myers Squibb and Merck & Co, which both reported downturns in the sales of blockbuster ant-PD-1 therapies, Opdivo and Keytruda, respectively, caused by the COVID-19 pandemic, with analysts taking a closer look. Also, AstraZeneca came out with results that pleased investors, as did Vertex Pharmaceuticals. 2 May 2021
Biotechnology
US biopharma company Vertex Pharmaceuticals has reported consolidated financial results for the first quarter of 2021 and reiterated full-year 2021 guidance for product revenues. 30 April 2021
Pharmaceutical
Rare forms of multiple sclerosis (MS) exhibit certain atypical imaging characteristics that are absent in typical MS, although the clinical features of these variants are similar to typical MS. 30 April 2021
Biotechnology
Shares in Protalix BioTherapeutics closed 44% lower on Wednesday after the firm and its partner Chiesi Farmaceutici announced receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) regarding the Biologics License Application (BLA) seeking accelerated approval of pegunigalsidase alfa (PRX–102) for Fabry disease. 29 April 2021
Biotechnology
French neurodegenerative diseases specialist Pharnext has announced new results from an interim analysis of an ongoing open-label follow-up extension study (‘PLEO-CMT-FU trial’) following the first double-blind, placebo-controlled Phase III study (PLEO-CMT trial) of PXT3003 for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). There is currently no approved drug treatment for CMT1A. 29 April 2021
Biotechnology
Nearly 18 months on from Enzyvant’s receipt of a Complete Response Letter (CRL) relating to its initial Biologics Licensing Application (BLA) for RVT-802, the company has filed an amended submission. 28 April 2021
Pharmaceutical
Shares of French pharma major Sanofi edged up 1.7% to 86.84 euros, after the company posted first-quarter 2021 financial results that were hit by currency aspects. 28 April 2021
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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