Positive data from Pfizer Inc. showed its gene therapy for treating adult men with moderately severe to severe hemophilia B hit the phase III Benegene-2 study’s primary endpoint. Fidanacogene elaparvovec, a vector containing an AAV capsid and a high-activity human coagulation factor IX (FIX), showed noninferiority and superiority in an annualized bleeding rate of total bleeds after its infusion vs. a prophylaxis regimen with FIX administered as part of usual care. The Pfizer results come little more than a month after the U.S. FDA gave the go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy, the first for treating adults 18 and older living with hemophilia B.

US spending bill spares animals, eases preclinical drug requirements

Tucked into the 4,155-page, $1.7 trillion spending bill for fiscal 2023 that U.S. President Joe Biden signed into law last week is a small provision that may have outsized impact on future biosimilar and other drug development. It also could vindicate Vanda Pharmaceuticals Inc.’s continued refusal to conduct additional animal studies on its investigational gastroparesis drug, tradipitant. Championed by lawmakers on both sides of the political spectrum, the provision modernizes the data that can be used to support drug development, including alternatives to animal studies – something the FDA has been hesitant to embrace in the past.

Japan gears up for another round of drug price revisions but stakeholders push back

Japan’s Central Social Insurance Medical Council (Chuikyo) said it would issue similar drug price revisions in 2023 as it did in 2022 for drugs listed on the National Health Insurance (NHI), confirming the move to annual price cuts on drugs. Drug price cuts issued by Japan’s Ministry of Health, Labour and Welfare (MHLW) in 2022 averaged about 6.6%, which translated to more than ¥600 billion (US$4.73 billion), and the pace of cuts is expected to accelerate. Price cuts in 2021 were worth roughly ¥430 billion. Japan’s Pharmaceutical Manufacturers Association, the Pharmaceutical Research and Manufacturers of America and the European Federation of Pharmaceutical Industries and Associations recommended that the 2023 NHI price revisions not be implemented due to the negative impact of five consecutive years of drug price cuts since 2018.

Regulatory news, COVID data and US approvals all down in 2022

U.S. FDA approvals in 2022 are down by 31.3% compared with last year and clearances for new molecular entities (NMEs) are at the bottom of all recent years.

Top Trends of 2022: Asia Pacific shakes off pandemic isolation, but focus remains on sovereign supply chains, regional deals

Investment in the fourth quarter of 2022 was dismal in Asia Pacific, as the global venture community focused on preserving capital. And the region faced other challenges throughout the year, as leaders in Australia and across Asia became acutely aware of the vulnerabilities in their supply chains. But the year also saw some big deals and collaborations involving companies across Asia Pacific, along with advances in regenerative and digital medicine. And, for the first time, in 2022, Chinese authorities allowed access for complete U.S. Public Company Accounting Oversight Board (PCAOB) audit inspections and investigations of PCAOB-registered public accounting firms headquartered in China and Hong Kong.

Also in the news

Amyris, Ardelyx, Cellectis, Hoth, Iveric, Junshi, Néovacs, Novavax, Opus Genetics, Pfizer, Pharnext, Transcode