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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
Intellia Therapeutics has announced Phase II results for its CRISPR-based therapy, NTLA-2002, aimed at providing a one-time treatment for hereditary angioedema (HAE). 25 October 2024
Biotechnology
Privately-held Swiss drug developer Neovii Pharmaceuticals has entered into an agreement with a unit of Shanghai Fosun Pharmaceutical, granting the latter an exclusive license to develop and commercialize Neovii's Grafalon (rabbit anti-human T-lymphocyte globulin, ATLG) in Chinese Mainland, Hong Kong SAR, Macau SAR and Taiwan region. 15 September 2022
Pharmaceutical
Coinciding with the 50th anniversary of its Lessines, Belgium, site, Japanese pharma major Takeda said it is investing close to 300 million euros ($304 million) in a new state-of-the-art production facility for the manufacturing of plasma-derived therapies for people with rare and complex chronic diseases. 15 September 2022
Biotechnology
Positive news from the medicines regulator yesterday saw US clinical-stage biotech Larimar Therapeutics’ shares close up 15.5% at $3.64. 15 September 2022
Biotechnology
Spanish CNS specialist Minoryx Therapeutics said today it has filed a Marketing Authorization Application (MAA) for its lead candidate leriglitazone to the European Medicines Agency (EMA) for the treatment of adult male patients with X-linked adrenoleukodystrophy (X-ALD). 14 September 2022
Pharmaceutical
Privately-held US developer of healthcare therapies Nacuity Pharmaceuticals and Icelandic firm Arctic Therapeutics International have entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of NPI-001 in Iceland for the treatment of hereditary cystatin C amyloid angiopathy (HCCAA). 14 September 2022
Pharmaceutical
The USA’s medicines cost-effectiveness watchdog the Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of treatment of amyotrophic lateral sclerosis (ALS). 13 September 2022
Biotechnology
Last week’s important news kicked off on Tuesday, when US biotech Sarepta Therapeutics got back on track with the development of its Duchenne muscular dystrophy (DMD) candidate SRP-5051 as the US Food and Drug Administration lifted its clinical hold on US clinical trials. There was more good news, this time for Amylyx, as an FDA advisory committee last Wednesday unexpectedly voted in favor of granting approval for its amyotrophic lateral sclerosis candidate AMX0035. Also of note, US biotech Iveric Bio raised its market capitalization by a third, after it announced positive Phase III data for its ophthalmic candidate Zimura. Swiss pharma giant Roche announced it was acquiring USA-based Good Therapeutics for $250 million. Also last week, Swiss firm Basilea Pharmaceutica entered into an asset purchase agreement with Nodus Oncology for its PARG inhibitor program, 11 September 2022
Biotechnology
Geneva-based cell-line company Selexis and NexImmune, a T cell specialist, has signed a service agreement to develop cell lines targeting rare cancers and autoimmune diseases. 9 September 2022
Biotechnology
US biotech Vertex Pharmaceuticals today announced that Jonathan Biller has been appointed chief legal officer, effective September 19, 2022. 8 September 2022
Biotechnology
Positive Phase III results have been presented on eplontersen, a ligand-conjugated antisense therapy from US RNA-targeted drug developer Ionis Pharmaceuticals and Anglo-Swedish pharma major AstraZeneca. 8 September 2022
Biotechnology
The US Food and Drug Administration has lifted a clinical hold on research into SRP-5051, a candidate under development by rare disease company Sarepta Therapeutics. 6 September 2022
Biotechnology
Important research news last week included Germany’s Bayer and US drug major Bristol Myers Squibb releasing new data at the European Society of Oncology (ESC) on their asundexian and milvexian, respectively. On the M&A front, last Tuesday Diurnal said it has agreed to an acquisition by US neurology specialist Neurocrine Biosciences, then on Thursday, Danish diabetes care giant Novo Nordisk announced a $1.1 billion deal to acquire Forma Therapeutics and its sickle cell disease drug candidate etavopivat. Also of note, Denmark’s Ascendis Pharma last week said it has filed for US Food and Drug Administration (FDA) approval for its hypoparathyroidism candidate TransCon PTH. 4 September 2022
Biosimilars
REMIDi4All launched yesterday with the aim of making a major leap forward in drug repurposing, Medicines for Europe, the trade body representing generics and biosimilar drugmakers, announced today. 2 September 2022
Biotechnology
The US Food and Drug Administration (FDA) has approved Spevigo (spesolimab-sbzo), the first approved treatment option for generalized pustular psoriasis s (GPP) flares in adults, developed by German family-owned pharma major Boehringer Ingelheim. 2 September 2022
Pharmaceutical
The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has announced its partnership with the Critical Path Institute (C-Path) to explore the feasibility of launching the Lysosomal Diseases Consortium, a public-private partnership. 2 September 2022
Pharmaceutical
The government of Russia has increased procurements of expensive drugs almost three-fold this year, mainly due the increase in the number of long-term agreements that are signed between the Russian state and drugmakers, reports The Pharma Letter’s local correspondent. 1 September 2022
Pharmaceutical
US journal JAMA Neurology has published positive results of the 24-week primary efficacy and safety analysis from the VISION-DMD study evaluating vamorolone, an investigational drug for the treatment Duchenne muscular dystrophy (DMD). 1 September 2022
Biotechnology
The US Food and Drug Administration (FDA) has approved French pharma major Sanofi’s Xenpozyme (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of the rare disease acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. 1 September 2022
Biotechnology
Australian biotechnology company CSL Limited has announced positive top-line Phase III results for garadacimab (CSL312), the company’s investigational first-in-class monoclonal antibody inhibiting Factor XIIa being developed as a long-term preventive treatment for patients with hereditary angioedema (HAE). 29 August 2022
Pharmaceutical
On the regulatory front last week, the long-awaited approval of Axsome Therapeutics’ Auvelity from the US Food and Drug Administration finally came through, clearing the drug for the treatment of major depressive disorder (MDD). Also, Foghorn Therapeutics announced that it has received a full clinical hold from the FDA regarding its Phase I trial of leukemia drug candidate FHD-286. And, Swiss drug developer Pharvaris last Monday revealed it had been notified of an FDA clinical hold on its PHA121 trial in hereditary angioedema. In the M&A space, a merger of eye care specialists is forthcoming, with last week’s announcement that Alcon has agreed to acquire Aerie Pharmaceuticals. Also of note, last Thursday Pfizer released positive new data on its respiratory syncytial virus (RSV) candidate RSVpreF. 28 August 2022
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