Rare Diseases
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Biotechnology
Japanese drugmaker Chugai, which is majority owned by Roche, On Friday revealed that Zenyaku Kogyo has obtained regulatory approval from the Ministry of Health, Labor and Welfare (MHLW), for an additional dosage and administration of an anti-CD20 monoclonal antibody Rituxan (rituximab) intravenous injection 100mg and 500mg, which is co-marketed by both companies, for chronic idiopathic thrombocytopenic purpura (ITP) in children. 23 November 2024
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
Biotechnology
Teijin Pharma, the core company of the Teijin Group’s healthcare business, announced today that it has acquired marketing approval for Revcovi (elapegademase [genetical recombination]) from the Ministry of Health, Labor and Welfare (MHLW). 26 March 2019
Pharmaceutical
The Australian government is providing almost A$25 million ($17.8 million) so Australians with cancer and rare diseases have access to clinical trials no matter where they live including regional and remote Victoria. 25 March 2019
Biotechnology
US biotech firm bluebird bio on Friday officially opened its first wholly-owned manufacturing facility in Durham, North Carolina, that will produce lentiviral vector for the company’s investigational gene and cell therapies. 25 March 2019
Pharmaceutical
Paroxysmal Nocturnal Hemoglobinuria (PNH) hit the news at the end of 2018 when the US Food and Drug Administration approved Ultomiris (ravulizumab-cwvz; Alexion) as a treatment for this very rare blood condition. This is ground-breaking news for a condition that is known to affect only one to nine people per million worldwide. 21 March 2019
Biotechnology
In another move by big pharma into gene therapy, US giant Pfizer has acquired a 15% equity interest in privately-held French gene therapy biotech Vivet Therapeutics and secured an exclusive option to acquire all outstanding shares. 21 March 2019
Biotechnology
The purse strings remain loose for Boston, USA-based Alexion Pharmaceuticals, with the announcement of two new pipeline-boosting collaborations in Europe. 21 March 2019
Pharmaceutical
A report has identified seven drugs launching this year that will reach blockbuster status by 2023. 20 March 2019
Biotechnology
Boston, USA-based biopharma Imara has closed an oversubscribed $63 million Series B cross-over financing. 18 March 2019
Biotechnology
The European Medicines Agency has granted approval for Roche to market MabThera (rituximab) against a rare condition, pemphigus vulgaris (PV). 15 March 2019
Biotechnology
X4 Pharmaceuticals, a clinical-stage biopharma company focused on treatments of rare diseases, today announced the completion of its merger with Arsanis, effective as of March 13, 2019. 14 March 2019
Biotechnology
Rare diseases specialist Strongbridge Biopharma has announced top-line findings from the extended evaluation phase of the Phase III SONICS study of Recorlev (levoketoconazole) for the potential treatment of endogenous Cushing’s syndrome. 11 March 2019
Pharmaceutical
In January of this year, the US Food and Drug Administration released updated draft guidance for industry entitled “Rare Diseases: Common Issues in Drug Development.” This replaces the previous version issued in 2015.1 7 March 2019
Pharmaceutical
US biopharma firm Ligand Pharmaceuticals says it has sold its Promacta (eltrombopag)-related intellectual property rights licensed to Novartis, including the royalty stream on worldwide net sales of Promacta to Royalty Pharma for $827 million in cash. 6 March 2019
Biotechnology
The US Food and Drug Administration has accepted the New Drug Application (NDA) for fedratinib and granted a Priority Review to the filing. 6 March 2019
Pharmaceutical
USA-based Corbus Pharmaceutical has announced the appointment of Craig Millian to the company as its first chief commercial officer ahead of the potential US Food and Drug Administration approval and commercial launch of its lead drug candidate lenabasum in 2021. 5 March 2019
Biotechnology
The growing interest in gene therapies was further confirmed by US Biotech major Biogen, which has reached an agreement to acquire a UK-based clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, at a price of $25.50 per share in cash. 4 March 2019
Pharmaceutical
Of interesting news last week, the US cost-effectiveness watchdog the ICER has issued an evidence report on Biogen’s Spinraza and Novartis’ yet-to-be-approved Zolgensma. M&A activity featured strongly last week, most notably with Swiss giant Roche’s decision to offer $4.3 billion to acquire US biotech Spark Therapeutics along with its hemophilia drug Luxturna and gene therapy pipeline, Also, France’s Ipsen plans to fork out ~$1.3 to buy USA-based Clementia Pharma and it rare disease drug palovarotene. Additionally, there were questions raised about Bristol-Myers Squibb’s previously-announced acquisition of Celgene, with two major institutional investors now raising their concerns about the proposed transaction. 3 March 2019
Pharmaceutical
At its last meeting in London before moving to new headquarters in Amsterdam as a result of Brexit, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) made positive recommendations for the approval of seven novel medicines. 1 March 2019
Biotechnology
US RNA-based drug developer Sarepta Therapeutics saw its shares gain 9.34% to $153.13 yesterday, after it announced it has exercised its option to acquire research partner Myonexus Therapeutics and new data on drug candidate MYO-101. 28 February 2019
Pharmaceutical
China will cut import value-added tax (VAT) from 16% to 3% on the first batch of rare disease drugs from March 1, according to the new policy of China’s National Medical Products Administration (NMPA), formerly known as CFDA, reports The Pharma Letter’s local correspondent, Wang Fangqing. 26 February 2019
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