| |JANUARY 20259CAPRICOR THERAPEUTICS SUBMITS BLA FOR FDA APPROVAL OF DERAMIOCEL IN DMDCENTRE EXTENDS GMP COMPLIANCE DEADLINE FOR SMALL PHARMA FIRMSCapricor Therapeutics, a biotech firm focused on developing innovative cell and exosome-based therapies for rare diseases, has announced the completion of the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for full approval of deramiocel, an investigational cell therapy aimed at treating patients with Duchenne muscular dystrophy (DMD) cardiomyopathy."The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD. This BLA is the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need," said Linda Marbán, Ph.D., chief executive officer of Capricor. "We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval."The complete filing of the rolling BLA was finalized as the company had earlier indicated in late December 2024 and is backed by Capricor's current cardiac findings from its phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, relative to natural history data from an FDA-funded and published dataset regarding the effects of DMD cardiomyopathy and possible biomarkers of disease progression. Capricor has sought a priority review, which, if approved, would shorten the review period from the usual 10 months to a prioritized 6-month timeline starting from when the FDA accepts the submission. POThe Centre intends to extend the deadline for complying with good manufacturing practice (GMP), aligned with World Health Organization (WHO) standards, for small pharmaceutical firms earning ?250 crore or below by one year to 31 December 2025.The regulation will be effective starting 1 January 2026. The previous deadline lapsed on 1 January 2025. This occurs against the backdrop of numerous representations made to the government by different associations asking for a timeline extension to help micro, small, and medium pharma companies adhere to the requirements of the updated Schedule M.Good Manufacturing Practices (GMP) are being enforced in the nation to enhance product quality through regulation of materials, methods, machinery, processes, staff, and the surrounding environment.The government announced that the new Schedule M was released in December 2023. To ensure a seamless transition from the current Schedule M to the updated Schedule M, a transition period of 6 months for large manufacturers (with revenue exceeding Rs.250 crore) and 12 months for MSMEs (with revenue below Rs.250 crore) was determined.India is a significant supplier of pharmaceuticals to developing nations, necessitating WHO GMP certification. Currently, there are approximately 2,000 units in the MSME category in the nation that have WHO GMP certification. India has approximately 10,500 drug manufacturing units, with about 8,500 classified as MSMEs. PO
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