With a $128 million series A financing, Diagonal Therapeutics Inc. launched to develop its lead program using agonist antibodies for treating, among other indications, the rare disease hereditary hemorrhagic telangiectasia. The antibodies are designed to activate a receptor complex in the TGF-β superfamily genetically impaired in patients with the bleeding disorder. Diagonal also is developing a treatment for the orphan disease pulmonary arterial hypertension.

Voydeya (danicopan), from Alexion, Astrazeneca Rare Disease, racked up its second global approval as the U.S. FDA greenlit it as an add-on therapy for treating extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH), a crowded market with several already approved treatments and more in development.

At first glance, the number of drugs that received accelerated approval from the U.S. FDA’s Center for Drug Evaluation and Research (CDER) in 2023 was nothing to write home about. Yes, CDER granted nine accelerated approvals last year, up from six in 2022. But the proportion of novel drugs with accelerated approval was 16% both years. And when compared with the 12 drugs in 2020 and the 14 that received accelerated approval in 2021, last year’s crop was a little lackluster. However, a deeper look at the 2023 class of accelerated approvals shows a historic milestone. For the first time since the path was created in 1992, the number of novel biologics getting accelerated approval at CDER outpaced the number of small-molecule drugs.

The U.S. FDA approved Orchard Therapeutics plc’s BLA for gene therapy atidarsagene autotemcel, making it the first treatment option for metachromatic leukodystrophy in the U.S. The one-time treatment, branded Lenmeldy, is indicated for children with presymptomatic late infantile, presymptomatic early juvenile or early symptomatic early juvenile disease.

Hun-taek Kim founded Tiumbio Co. Ltd. in 2016 after spending more than two decades at a major chemical and life science firm, SK Chemicals Co. Ltd. “The prospects for our three major assets are very bright, and the probability of failure is low,” CEO Kim told BioWorld.

The new director of the U.S. NIH, Monica Bertagnolli, has set out the prospectus for her tenure, saying she intends to apply the agency’s $47 billion per annum budget to reverse the decline in health and life expectancy in the U.S.

Hun-taek Kim founded Tiumbio Co. Ltd. in 2016 after spending more than two decades at a major chemical and life science firm, SK Chemicals Co. Ltd. “The prospects for our three major assets are very bright, and the probability of failure is low,” CEO Kim told BioWorld. “We’re looking for a breakthrough in rare diseases – to develop new treatments for [niche] markets with large unmet demand.”

Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.

Based on positive phase III study data, Applied Therapeutics Inc. plans to take its CNS-penetrant aldose reductase inhibitor to the U.S. FDA to talk about an NDA for treating the rare disease sorbitol dehydrogenase (SORD) deficiency. Interim data from 12 months of treatment showed govorestat (AT-007) hit the study’s primary endpoints along with several key secondary endpoints. The double-blind, placebo-controlled registrational study of patients ages 16 to 55 is ongoing, with another 12 months of data yet to come. SORD, a hereditary axonal neuropathy created by sorbitol dehydrogenase gene mutations, affects about 3,300 people in the U.S. and about 4,000 in Europe, according to Applied Therapeutics.