Biomarin Pharmaceutical Inc.’s data supporting the use of Voxzogo (vosoritide) in children with the most common form of dwarfism proved compelling enough for the FDA, which cleared the modified C-type natriuretic peptide as the first treatment for achondroplasia, a rare genetic disease affecting bone growth. Voxzogo was approved for children ages 5 and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow. There are more than 10,000 children in the U.S. who meet the criteria and about 25% of them have open growth plates. Voxzogo, an analogue of a C-type natriuretic peptide, directly targets the underlying pathophysiology of achondroplasia by down-regulating FGFR3 signaling, which, in turn, promotes endochondral bone formation. The approval came a day ahead of its original Nov. 20 PDUFA date under an accelerated approval pathway.

Sotio places billion dollar bet on Legochem’s ADC tech

Sotio Biotech AS has licensed Legochem Biosciences Inc.’s technology for five new antibody-drug conjugates (ADC) in a deal that could bring the latter more than $1 billion. Sotio will have the rights to deploy Legochem’s Conjuall and potent linker-payload platform, including multiple different payloads, for five therapeutic programs targeting solid tumors and combine its proprietary antibodies with it.

COVID-19 boosters cleared by FDA for everyone 18 and up

The FDA amended the emergency use authorizations for the Moderna Inc. COVID-19 vaccine as well as the shot from Pfizer Inc. and Biontech SE. A single booster dose was cleared for people 18 years of age and older in the U.S. The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices is meeting today to discuss possible further guidance.

Clinical news slows, but still up 7% over last year

Efforts to find new treatments for cancer and infectious diseases account for nearly half of the phase I, II and III clinical data recorded in 2021. BioWorld has tracked 3,633 clinical entries this year, including 1,381 for phase I, 1,313 for phase II, and 939 for phase III. The total is about 7% more than the same period of 2020, which had 3,391 entries, with most of the growth seen with phase I and phase II data. While it is a busy year for clinical news, the flow began to slow down mid-year. In April, for example, the gap with last year indicated that 2021 was 26% ahead. The largest portion of the clinical news this year is for the therapeutic areas of oncology (27.91%) and infectious diseases (20.78%), including COVID-19.

Connect sees positive readout in phase II AD trial, but Dupixent comps a mystery for now

A phase II trial of Connect Biopharma Holdings Ltd.'s CBP-201 in moderate-to-severe atopic dermatitis (AD) showed all three doses tested reduced eczema severity vs. placebo by a statistically significant percentage at 16 weeks, meeting the trial's primary efficacy endpoint. Nonetheless, analysts seeking numerical details to fuel comparisons with blockbuster AD therapy Dupixent (dupilumab, Sanofi SA and Regeneron Pharmaceutical Inc.) were left short, leaving the question of whether '201 can differentiate from the blockbuster a mystery. Connect's shares (NASDAQ:CNTB) fell 54% by midday following the news, even as co-founder and CEO Zheng Wei shared plans to start a phase III trial program in mid-2022.

Conditioning agents in HSCT in the works at multiple sites

New conditioning agents for hematopoietic stem cell transplant (HSCT) – intravenous infusion in order to crank up production of blood cells in patients whose bone marrow or immune system is damaged or defective – are in the works at a handful of companies, with Jasper Therapeutics Inc. ushering a distinctive candidate through the pipeline. JSP-191, an anti-CD117 monoclonal antibody, is designed (unlike other candidates) to block the stem cell factor signaling pathway. Others in the space include Actinium Pharmaceuticals Inc., Gilead Sciences Inc., Magenta Therapeutics Inc. and Molecular Templates Inc.

COVID-19 hits UK’s research charities where it hurts: funding

LONDON – Medical research charities that form an integral part of the U.K. health research system are still reeling from the impact of COVID-19 and now expect it will be three years before funding returns to pre-pandemic levels.

Also in the news

60 Degrees, AB Science, Antengene, Apellis, Arena, Ascendis, Biogen, Biontech, Bridgebio, Bristol Myers Squibb, Cali, Cellectis, Cellevolve, Chimerix, Connect, Curevac, Cytodyn, Cytovia, Dermata, Enanta, Enterin, Gilead, Gracell, Helsinn, Immunic, Immutep, Innovation, Ionis, Jaguar Health, Kancera, Locanabio, Merck, Moderna, Oncolys, Optinose, Pfizer, Plus, Rain, Redhill, Roche, Scancell, Schrödinger, Second Genome, Shenzhen, Tavotek, Todos Medical, UCB, Vaxart, Vaxxinity, Voyager