News of a partial FDA clinical hold for pediatric patients after safety reports from an ongoing trial testing palovarotene in fibrodysplasia ossificans progressiva (FOP) identified cases of early growth plate closure sent shares of Ipsen SA (Paris:IPN) falling 13.6% Friday.

Nearly a month ahead of the PDUFA date, red blood cell maturation drug luspatercept cleared the FDA for treating anemia in adults with beta-thalassemia who require regular red blood cell (RBC) transfusions. Branded Reblozyl, the drug, developed in a collaboration between Celgene Corp. and Acceleron Pharma Inc., is expected to be available in one week following approval.

It's been a busy September for Ardelyx Inc. so far. Less than two weeks after the Fremont, Calif-based company reported promising pivotal data for tenapanor in chronic kidney disease patients with hyperphosphatemia, the sodium hydrogen exchanger 2 (NHE3) inhibitor won FDA approval for use in irritable bowel syndrome with constipation (IBS-C).

The convergence of artificial intelligence (AI) and the health care sector was inevitable. The advent of machine-learning and deep-learning technologies capable of analyzing and synthesizing massive amounts of data with algorithms designed to mimic human-level decision-making seems a natural fit for an industry in dire need of greater efficiency.

PHILADELPHIA – Compared to oncology, chronic diseases such as cardiovascular disease, diabetes and dementia represent much more substantial costs to the overall U.S. health care system, yet investment and R&D innovation in those areas has been on a steady decline over the past decade or so, according to research compiled by the Biotechnology Innovation Organization (BIO).

Tocagen Inc.'s disclosure that the independent data monitoring board for its ongoing phase III trial in recurrent high-grade glioma (HGG) recommended the study continue without modification following a planned interim analysis drew mixed reactions from analysts and sent shares of the San Diego-based company (NASDAQ:TOCA) falling 38% Wednesday.

Shares of Solid Biosciences Inc. took it on the chin again, after the company reported a serious adverse event (SAE) from the second cohort of its phase I/II Ignite DMD study testing gene therapy SGT-001 in Duchenne muscular dystrophy. The latest setback comes three months after Cambridge, Mass.-based Solid disclosed disappointing data from the first patient cohort and a year after the FDA lifted a clinical hold placed on the same trial after the first patient treated suffered an SAE.

Shares of Catalyst Pharmaceuticals Inc. tumbled 40% Tuesday on word that the FDA had cleared Ruzurgi (amifampridine) for treating pediatric patients with Lambert-Eaton myasthenic syndrome (LEMS). Catalyst, of Coral Gables, Fla., currently markets its LEMS treatment, Firdapse (amifampridine phosphate), for adult patients but has come under congressional scrutiny for its hefty $375,000 annual price tag, particularly since the active drug was once available to patients for free.

VIENNA – After nearly three days of partnering meetings and sessions touting the latest therapeutic advances such as cell and gene therapy and the microbiome, along with presentations depicting the unprecedented level of capital flowing into the sector, it would be easy to think the biopharma industry had reached optimal capacity. But as the BIO Europe Spring meeting came to a close Wednesday, Evotec AG CEO and Austria native Werner Lanthaler suggested that progress and innovation "aren't happening as fast as they should."