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. Read More
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. Read More
Chinese biotechs Biotheus Inc. and Hansoh Pharmaceutical Group Co. Ltd. partnered again under a new potential ¥5 billion (US$698.98 million) deal to develop bispecific antibody-drug conjugates (BsADCs), using Biotheus’ EGFR-cMet bispecific antibody. Read More
Fennec Pharmaceuticals Inc. has out-licensed its chemoprotective formulation of sodium thiosulfate, Pedmarqsi, to Norgine BV for €40 million (US$44 million) up front and up to an additional €210 million (US$229 million) in commercial and regulatory milestones. The injectable drug reduces the risk of cisplatin-induced ototoxicity in pediatric patients 1 month and older with localized, nonmetastatic solid tumors. Read More
PYC Therapeutics raised AU$40 million of an anticipated AU$74 million (US$48.6 million) capital raise to advance three candidates, including lead candidate VP-001, which could potentially be the first treatment for retinitis pigmentosa type 11 (RP11), which causes blindness that begins in childhood and ultimately leads to legal blindness by middle age. Read More
Techbio specialist Relation Therapeutics Ltd has raised $35 million in new seed funding, bringing total seed money to $60 million, as it advances development of its in silico/wet lab platform for identifying drug targets in the non-coding parts of the genome. The company is building a “lab in the loop” system where in depth ‘omics profiles of single cells from fresh patient tissues are analyzed by its machine learning engine to uncover the genetic basis of clinical phenotypes and identify novel targets. Read More
Aignostics GmbH has embarked on raising a €20-€30 million (US$21.74-$32.61 million) series B round after validating its artificial intelligence (AI)-driven precision oncology platform by signing a co-development deal with Bayer AG. This is the first pharma partnership for the Berlin, Germany-based company. Financial terms were not disclosed, but Aignostics will receive an up-front payment and is eligible to receive success-based milestone payments and royalties on any commercialized therapies that result from the collaboration. Read More
The U.S. Court of Appeals for the Third Circuit may have ruled last year that the Department of Health and Human Services doesn’t get to fill in the gaps in the law that created the 340B prescription drug discount program, but some states and lawmakers are coming up with their own workarounds to force drug manufacturers to give the discounts to an unlimited number of contract pharmacies. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aquestive, Auxilius, Cybin, Jasper, Lantern, Merck, Nuvation, Recce. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Adaptive Phage, Astellas, Audentes, Cartesian, Biomx, Bristol Myers Squibb, Gilgamesh, Idorsia, Karuna, Viatris. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Beigene, Bristol Myers Squibb, Immvira, Madrigal. Read More
