The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5.
While the biopharma industry was widely praised for its fast response to the COVID-19 pandemic, moves are afoot to ensure that the world is better prepared in case another pandemic hits. Moderna Inc. was one of the companies that blazed a trail in the early stages of the pandemic with its revolutionary mRNA vaccine. Now the firm is investing in manufacturing and R&D in the U.K. to make good on a pledge to respond to the next global disease threat within 100 days of its detection.
In the last decade, Bayer AG and Novartis AG have shown that radiopharmaceutical technology can produce marketable drugs, with Xofigo (radium-223 dichloride) and Lutathera (lutetium [177Lu] oxodotreotide), respectively approved for prostate cancer and neuroendocrine tumors.
Valneva SE has struggled to get its COVID-19 vaccine on the market, but efforts to get the first Lyme disease vaccine approved in years now appear firmly on track after partner Pfizer Inc. invested €90.5 million (US$94.9 million) in the project. New York-based Pfizer has been working with Saint-Herblain, France based Valneva on the Lyme disease vaccine since April 2020, when the big pharma bought into the development program for the vaccine candidate codenamed VLA-15 for $130 million.
Alzheimer’s experts have suggested that future research may involve combination therapies after yet another trial failure involving a drug targeting amyloid – in this case Roche Holding AG’s crenezumab. Originally discovered by Swiss biotech AC Immune SA, of Lausanne, crenezumab failed to slow or prevent cognitive decline in people with a certain mutation that causes early onset in a closely watched trial.
Sanofi SA and GSK plc were beat to market during the first wave of COVID-19 by vaccines from upstarts such as Biontech SE and Moderna Inc. – but the French and U.K. vaccine specialists are gaining traction in the race to develop booster shots against newer variants.
GSK plc is preparing filings for its respiratory syncytial virus (RSV) vaccine in older people following supportive late-stage trial results, a product which could play a vital role as it spins out its consumer business arm. The vaccine could be a potential blockbuster for GSK, worth as much as $7 billion in peak annual sales if approved in all its potential uses – although trials in pregnant women remain on hold because of safety concerns.
In August 2020, it looked like DBV Technologies SA was in considerable trouble after the U.S. FDA served it with a rejection for its Viaskin Peanut allergy patch, raising concerns efficacy could be compromised because the product wasn’t sticking to the skin well. But could the Montrouge, France-based firm be making a comeback with the epicutaneous technology?
Philadelphia’s Mineralys Therapeutics Inc. has secured a further $118 million to develop MLS-101, a potential hypertension drug in-licensed from Mitsubishi Tanabe Pharma Corp.
Could Merus NV be on course to get the fourth “tumor agnostic” drug approved, a groundbreaking approach to cancer that focuses on mutations rather than location of the disease? According to a trial update revealed at the American Society of Clinical Oncology (ASCO) conference the biotech from Utrecht, the Netherlands, could join Merck & Co Inc., Bayer AG/Eli Lilly and Co. and Roche Holding AG with its early-to-mid stage candidate, zenocutuzumab.
