A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy.
Shares in France’s vaccine specialist Valneva SA were sharply down after the U.K. government tore up a contract to supply 100 million doses of its COVID-19 vaccine, codenamed VLA-2001. Until today, Valneva was one of the largest COVID-19 vaccine contractors with the U.K. government, which got its orders early in the pandemic. Valneva was supposed to be supplying 100 million doses of its inactivated virus vaccine to the U.K., which had an option on a further 90 million shots.
It was a busy week for the U.K.’s drugs regulator, the Medicines and Healthcare Products Regulatory Authority (MHRA), which fast tracked decisions on novel drugs for cancer and eczema and made a ruling on booster COVID-19 jabs from Astrazeneca plc and Pfizer Inc. But even as Prime Minister Boris Johnson tweeted his support over the approval of Amgen Inc.’s cancer drug Lumykras, there are questions over the future of the regulator as it faces a round of job cuts because of a lack of funding following Brexit.
Astrazeneca plc is eyeing an addition to its respiratory diseases portfolio after a potential first-in-class “rescue” asthma inhaler, PT-027, met its goals in two phase III trials.
Diaccurate SAB has scooped a cancer drug from Germany’s Merck KGaA, transforming the small French biotech into a clinical-stage company. With expertise on its scientific advisory board from Nobel prize winner Tasuku Honjo, famously credited with the work establishing PD-1 as a target in immune-oncology, Diaccurate said it has fought off other competitors to acquire M-2698.
Sanofi SA has added to its general medicines portfolio with a $1.9 billion acquisition of Kadmon Holding Inc. and its recently-approved graft-vs.-host disease drug Rezurock (belumosudil).
The FDA has put a hold on a clinical study of a rare disease gene therapy BMN-307 from Biomarin Pharmaceutical Inc. after several mice developed liver tumors following a high dose in a preclinical trial.
The European Commission and Astrazeneca plc have ended their legal row over COVID-19 vaccines, while also reaching an agreement over supplies of remaining shots to Europe. The settlement brings to an end an unseemly row over vaccine supply, which further soured relations between the EU and U.K. following Brexit.
The board of rare disease specialist Swedish Orphan Biovitrum AB has accepted a takeover bid from the U.S. private equity firm Advent International and Aurora Investment, an affiliate of capital markets group GIC, valuing it at about SEK69.4 billion (US$8 billion).
The race to develop better antivirals for COVID-19 is heating up after Pfizer Inc. and Merck & Co. Inc. pushed ahead with clinical development of late-stage candidates, while Rigel Pharmaceuticals Inc. announced encouraging midstage data.
