Rare Diseases
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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
French drugmaker Sanofi has revealed the results from the Phase III LUNA 3 trial of rilzabrutinib, an oral bruton tyrosine kinase (BTK) inhibitor, in persistent or chronic immune thrombocytopenia (ITP). 6 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, under development by Ionis Pharmaceuticals. 4 November 2024
Pharmaceutical
Disc Medicine today announced a successful End of Phase 2 with the US Food and Drug Administration (FDA) supporting the regulatory path forward, including potential for accelerated approval, for bitopertin in erythropoietic protoporphyria (EPP). 4 November 2024
Pharmaceutical
On Friday, the US Food and Drug Administration published the FDA Voices: “FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub,” by Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research (CDER) and Dr Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER). 4 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted a Rare Pediatric disease designation for BPM31510T, an investigational treatment for epidermolysis (EB) from BPGbio. 4 November 2024
article
The European Union's antitrust enforcer hit Israeli drugmaker Teva Pharmaceutical Industries (NYSE: TEVA) with a 463 million-euro ($502 million) fine Thursday for launching a smear campaign against a rival multiple sclerosis drug and misusing the patent system to thwart the competitor's attempt to entering the market. 31 October 2024
Biotechnology
US biotech major Amgen late yesterday released financial results for the third quarter of 2024, showing strong sales and earnings growth. 31 October 2024
Pharmaceutical
Italy’s Chiesi has announced the publication of results from the Phase III BRIGHT study of pegunigalsidase alfa 2mg/kg, administered every four weeks for 52 weeks, in adults with Fabry disease who were previously treated with agalsidase alfa or beta, administered every two weeks. 30 October 2024
Biotechnology
US biotech Biogenraised its annual earnings guidance and reported better-than-expected top and bottom lines for the third quarter of 2024, sending the company’s shares rising 3% in pre-market trading Wednesday. 30 October 2024
Biotechnology
Swedish Orphan Biovitrum, the Nordic biotech also known as Sobi, is presenting new research on the use of emapalumab in patients with macrophage activation syndrome (MAS) in Still’s disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset still’s disease (AOSD) who had an inadequate response to high-dose glucocorticoids (GCs). 30 October 2024
Pharmaceutical
UK pharma major AstraZeneca today announced that the National Institute for Health and Care Excellence (NICE) published its final draft guidance (FDG) recommending Wainzua (eplontersen) as a treatment option for hereditary transthyretin-related amyloidosis (ATTR) in adults with stage 1 or stage 2 polyneuropathy across England and Wales. 29 October 2024
Biotechnology
AstraZeneca’s Fasenra (benralizumab) has been approved in the European Union (EU) as an add-on treatment for adult patients with relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). 28 October 2024
Biotechnology
California’s Vera Therapeutics has released 96-week follow-up results from its ORIGIN Phase IIb trial of atacicept, targeting IgA nephropathy (IgAN). 28 October 2024
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
Biotechnology
AveXis has reacted to the release on Friday of a report assessing the likely cost-effectiveness of the company’s investigational spinal muscular atrophy (SMA) drug Zolgensma (onasemnogene abeparvovec- xioi), as well as slamming the price of Biogen’s already marketed Spinraza (nusinersen). 26 February 2019
Biotechnology
The Institute for Clinical and Economic Review (ICER) has given its verdict on what constitutes a fair US price for two drugs treating spinal muscular atrophy (SMA), an inherited neurodegenerative disease that means many babies never reach their second birthday. 25 February 2019
Biotechnology
French drugmaker Ipsen has entered into an agreement to acquire Canada-headquartered biotech Clementia Pharmaceuticals. 25 February 2019
Biotechnology
Results from the SYROS study demonstrate that long-term treatment with idebenone consistently reduced the rate of respiratory function loss in patients with Duchenne muscular dystrophy (DMD) for up to six years in a real-world setting. T 25 February 2019
Biotechnology
The UK’s medicines cost-effectiveness watchdog has published draft guidance which confirms its earlier decision not to recommend Brineura (cerliponase alfa), made by US biotech firm BioMarin Pharmaceutical, for children with Batten disease – a very rare inherited condition affecting between one and six babies each year in the UK. 22 February 2019
Generics
Swiss generic drugmaker Dipharma today announced that the European Commission has granted Marketing Authorization of its generic miglustat. 20 February 2019
Pharmaceutical
Cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) has been encouraged to change the way it assesses applications to gain reimbursement for rare disease medicines in England. 19 February 2019
Biotechnology
Product sales at Shire grew by 4% to $15 billion in its last year as a standalone company. 15 February 2019
Pharmaceutical
Recordati went through a change in its majority ownership last year, with private equity fund CVC Capital Partners paying three billion euros ($3.4 billion) for nearly 52% in Fimei, the Recordati family holding company. 11 February 2019
Biotechnology
An ongoing open-label extension study treating patients with brain disease drug Brineura (cerliponase alfa) continued to show a reduced rate of decline compared to a natural history cohort of CLN2 disease for three years as measured by the CLN2 Clinical Rating Scale. 11 February 2019
Biotechnology
The US Food and Drug Administration (FDA) has extended the review period for the supplemental New Drug Application (sNDA) for Jakafi (ruxolitinib) for the treatment of patients with acute graft-versus-host disease (GVHD) who have had an inadequate response to corticosteroids. 8 February 2019
Biotechnology
Within a day of announcing positive Phase III findings on its multiple myeloma candidate isatuximab, Sanofi has received approval from the US Food and Drug Administration (FDA) for another of its innovative assets. 7 February 2019
Pharmaceutical
French pharma major Sanofi has been boosted by a positive end of year financial results statement, showing the firm took in 8.9 billion euros ($10.1 billion) in the final three months of the year, an increase of 3.5%, with sales growth driven by its specialty care and vaccine units. 7 February 2019
Pharmaceutical
Daiichi Sankyo announced that the US Food and Drug Administration has accepted a New Drug Application (NDA) and granted priority review for pexidartinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT). 6 February 2019
Pharmaceutical
Italian drugmaker Recordati has signed a license agreement with Aegerion Pharmaceuticals, a subsidiary of Novelion Therapeutics for the exclusive rights to commercialize Juxtapid (lomitapide). 6 February 2019
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