Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

The FDA gave full approval based on a Phase II study, without the need for Phase III confirmatory data, for Amylyx’s ALS drug Relyvrio but the $158,000 WAC could spark a debate over pricing.

Japan has granted to the first approval worldwide to Daiichi Sankyo’s dual EZH1/2 inhibitor valemetostat, for the treatment of adult T-cell leukemia/lymphoma. Taisho’s trivalent anti-TNFα nanobody ozoralizumab also received its global first approval, as part a large new batch of regulatory nods in the country.

The Dutch firm’s US filing for its first-in-class PI3K inhibitor has been accepted under priority review, bringing the asset closer to becoming the first disease-modifying option for a rare, genetic disorder.

Special guest, Citeline's Annie Siu, discusses the recent ESMO meeting and Jiangsu Hengrui and BeiGene's PD-1 data for hepatocellular carcinoma in this latest episode of the Chinese-language China Biotech podcast.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

After the Aduhelm controversy, Biogen has another neurodegenerative disease drug which so far can only show efficacy against surrogate biomarkers – will the FDA be swayed again this time?

Discussion of the launch trajectory for the twice-yearly cholesterol therapy dominated the marketing portion of Novartis’s investor event and the firm is confident the foundations have been laid to push strong sales growth by the second half of next year.

After successive CRLs in 2020 and 2021, the drug gained a broad label for hearing loss in children treated with cisplatin for localized, non-metastatic solid tumors.

With a European approval in the bag for Tezspire, AstraZeneca is looking for quick and successful launches of the severe asthma drug so it can mount a challenge to the other biologics in the space, notably Sanofi and Regeneron’s Dupixent.

The $3m gene therapy for the rare disease cerebral adrenoleukodystrophy marks bluebird’s second approval in just under a month.

After highlighting oncology drugs pegged to make a splash next year with the help of Biomedtracker, Scrip takes a look at seven potential blockbusters across a range of therapeutic areas including neurology, dermatology and blood disorders slated for approval next year.