Solid Biosciences Inc. has announced approvals of its IND application and CTA by the U.S. FDA and Health Canada, respectively, for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by mutations in the RYR2 and CASQ2 genes.
Insightec Ltd. received U.S. FDA approval for use of its Exablate Neuro device to address severe motor symptoms in patients with Parkinson’s disease, offering a new option for patients who have not found adequate relief from medications. Exablate Neuro uses focused ultrasound to create lesions in the brain without requiring an incision or implant.
Patent reform in the U.S. revolves largely around the subject matter eligibility question, but Congress is reluctant to intervene – a predicament addressed recently by Andrei Iancu, formerly the director of the U.S. Patent and Trademark Office.
The U.S. Centers for Medicare & Medicaid Services indicated it will act on a previous proposal to terminate the Treatment Choices Model for ESRD at the end of the current calendar year because of a failure of the program to deliver on the promised efficiencies and improvement in outcomes.
China’s National Medical Products Administration gave the green light to Simcere Pharmaceutical Group Ltd.’s Enzeshu (suvemcitug) for treating recurrent ovarian cancer, fallopian tube cancer, or primary peritoneal cancer in combination with paclitaxel, liposomal doxorubicin, or topotecan in adults who have received at least one systemic therapy after platinum resistance.
Four days ahead of its July 7 PDUFA date, the U.S. FDA granted accelerated approval to Dizal (Jiangsu) Pharmaceutical Co. Ltd.’s sunvozertinib (DZD-9008), branded as Zegfrovy, for treating adults with locally advanced or metastatic non-small-cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations following platinum-based chemotherapy.
Wuhan Healthgen Biotechnology Co. Ltd. gained clearance from the Shanghai Stock Exchange July 1 to list under a recently reinstated IPO growth tier geared towards supporting “unprofitable” biotechnology firms.
Representatives of patients’ groups, industry bodies and venture philanthropy funders are calling for a renewal of the U.K. Rare Diseases Framework, to put fresh momentum behind translational research and clinical trials, streamline regulatory oversight and improve access to therapies.
Led by the American Academy of Pediatrics, several medical groups went to court July 7 to force Health and Human Services Secretary Robert Kennedy to restore the CDC’s COVID-19 vaccine recommendations for pregnant women and healthy children ages 6 months to 17 years.
Boston Scientific Corp. gained a second U.S. FDA approval for its Farapulse pulsed field ablation catheter, expanding its use into drug-refractory, symptomatic persistent atrial fibrillation (AF) in addition to its existing approval for paroxysmal AF. The expanded indication marks the first of several regulatory approvals the company expects in the coming months.
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