Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full length dystrophin in Becker muscular dystrophy.
Pulsesight Therapeutics SAS has submitted a clinical trial authorization (CTA) to the French authority ANSM seeking to conduct a first-in-human phase I trial of PST-611 for dry age-related macular degeneration (AMD)/geographic atrophy (GA).
The U.S. FDA’s Accreditation Scheme for Conformity Assessment (ASCA) program has its benefits, but industry had some sharp words for the agency in commenting on the 2024 ASCA draft guidance. The Advanced Medical Technology Association stated that FDA reviewers asked for full conformity test results for roughly 5% of premarket applications, a rate seen as excessive given the program’s design.
The U.S. FDA posted a draft guidance that would revisit a 2014 final guidance on sex-specific data drawn from medical device clinical trials, expanding the scope to include considerations of gender.
Medtronic plc secured CE mark approval under the EU Medical Device Regulation for its Brainsense adaptive deep brain stimulation system. The technology represents a major advancement in the treatment of Parkinson’s disease as it provides real-time, self-adjusting brain stimulation based on the patient’s brain activity, offering a more personalized therapy.
CSPC Pharmaceutical Group Ltd. has obtained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYS-6041, an antibody-drug conjugate, for advanced solid tumors.
Data from two out of three positive studies faced the U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee as members examined the package submitted by Seikagaku Corp., of Tokyo, for SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia.
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
The U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee will scrutinize Jan. 10 the safety and efficacy of Ferring Pharmaceuticals Inc.’s SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia.
It’s taken nearly a decade for the U.S. FDA to go from zero to 60 in approving biosimilars. Currently, 63 biosimilars have been approved in the U.S., thanks to 18 new approvals in 2024 that stretched the number of biologics referenced by biosimilars from 14 to 17. That’s an all-time record, CDER Director Patrizia Cavazzoni said, as she released the drug center’s annual approval report for 2024.
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