Where European regulatory decisions were concerned, there was good news and bad news for pharma today as Sanofi SA and Regeneron Pharmaceuticals Inc. got the go-ahead for expanded approval of Dupixent (dupilumab) in pediatric atopic dermatitis patients whilst Ipsen SA’s ultra-rare bone disease drug palovarotene was left off the shelf as efficacy data failed to impress. The decision by the European Medicine Agency’s Committee for Medicinal Products for Human Use not to approve the targeted, oral retinoic-acid receptor gamma agonist palovarotene for the treatment of fibrodysplasia ossificans progressiva was based on data from the phase III MOVE trial.

Parties big and small forge ahead with ROR1 research 

Receptor tyrosine kinase like orphan receptor 1 (ROR1) continues to intrigue drug developers, with a handful of parties known to be in play. Among them is Caribou Biosciences Inc., which last December said it had chosen ROR1 as the target for CB-020, an induced pluripotent stem cell-derived allogeneic CAR-NK cell therapy for solid tumors. ROR1 has piqued the interest of big pharma, too, which signed a couple of sizeable deals in 2020.

Australia begins first independent review of its health technology assessment process 

The Australian government has begun its closely watched independent review of the health technology assessment (HTA) process in the country, and it delivered a first peek at what it will consider. This independent review of the HTA system is the first of its kind in nearly 30 years. A 2021 parliamentary report recommended significant reforms to the nation’s health care system to ensure Australians have faster access to new drugs and devices. The report featured 31 recommendations that included streamlining the HTA process, particularly for cell and gene therapies.

Bipartisan members of US Senate ink bill for FDA-PTO cooperation

Four members of the U.S. Senate have inked a draft bill that would require the FDA and the Patent and Trademark Office to set up a task force designed to improve communication between the two agencies. This would appear to replicate a bill introduced during the 117th Congress, but not ultimately passed, and there is little clarity this early in the legislative cycle as to the prospects for this latest iteration.

The 2022 UK Biotech Financing report: IPOs dropped but venture cap stood strong

It’s been a tough year for raising biotech money, according to the new U.K. Biotech Financing Report. In the past year, IPOs in the U.K. raised £28 million (US$34.65 million), the lowest annual total in a decade. New launches were a rarity, the report noted, with £1.8 billion (US$2.23 billion) raised by U.K. biotechs in 2022, a drop from the £4.5 billion (US$5.57 billion) in 2021. There were bright spots, however, including venture capital investment of £1.2 billion (US$1.49 billion), the third-best year on record for the U.K. U.S. companies increased their acquisitions of U.K. companies, with the report citing Pfizer Inc.’s purchase of Reviral Ltd. for £420 million (US$520 million), Gilead Sciences Inc.’s acquisition of Mirobio Ltd for £305 million (US$377 million) and Abbvie Inc.'s purchase of DJS Antibodies Ltd. for £229 million (US$283 million). The annual report is produced by Clarivate, which publishes BioWorld, and the U.K.’s Bioindustry Association, a trade association representing nearly 500 companies.

US GAO report highlights growing use of third-party funding in product liability litigation 

Makers of medical devices and pharmaceuticals face a significant risk of product liability litigation, but the use of third-party funding of such lawsuits is a novelty in the U.S. relative to some other Western nations. Nonetheless, a new report by the U.S. Government Accountability Office (GAO) makes clear that third-party litigation funding is an increasingly common practice that seeks to include uninjured parties in mass tort litigation, thus endangering the fortunes of those who invest in life science companies.

Also in the news

AC Immune, Acerus, Altamira, Aridis, Ascletis, Biocryst, Bristol Myers Squibb, Celularity, Centessa, Daewoong, Daichi Sankyo, Faron, F-Star, Fzata, Hemogenyx, Immusoft, Invox, Ivexsol, Lapix, Lighthouse, Liminal, Magenta, Netris, Novavax, Ocuphire, Orbsen, Oxurion, Quince, Sanofi, Senti, Siva, Sona, Syros, Tetra, Valeo