Sarepta Therapeutics has received accelerated approval from the FDA for Elevidys (delandistrogene moxeparvovec), a gene therapy for treating ambulatory pediatric patients aged 4-5 with Duchenne muscular dystrophy (DMD). This approval is based on micro-dystrophin expression observed in patients treated with Elevidys, with continued approval contingent on verifying clinical benefit in confirmatory trials.
Sarepta's Elevidys Approval Details
The accelerated approval of Elevidys, an adeno-associated virus-based gene therapy, follows a somewhat contentious review process, with the FDA's Cellular, Tissue and Gene Therapies Advisory Committee initially voting 8:6 in favor of approval. Concerns were raised regarding unambiguous evidence of clinical benefit in DMD patients. The FDA has indicated that a non-age restricted expansion to SRP-9001 will be granted provided the phase III EMBARK study achieves its objectives. The EMBARK trial, a global, randomized, double-blind, placebo-controlled phase III study, is fully enrolled, with top-line results expected in late 2023. This trial will serve as the post-marketing confirmatory trial.
Bristol Myers Squibb's Camzyos Approved in EU for HCM
Bristol Myers Squibb (BMY) announced that the European Commission has approved Camzyos (mavacamten) for treating symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (HCM) in adult patients. Camzyos is the first and only cardiac myosin inhibitor approved in the European Union.
The approval is based on positive efficacy and safety results from the EXPLORER-HCM and VALOR-HCM phase III studies. Both studies met all primary and secondary endpoints, demonstrating improvements in exercise capacity and symptom burden. Camzyos is already approved in the United States for treating adults with symptomatic NYHA class II-III HCM to improve functional capacity and symptoms.
FibroGen's Pamrevlumab Fails in IPF Trial
FibroGen, Inc. experienced a setback as its late-stage study, ZEPHYRUS-1, evaluating pamrevlumab in patients with idiopathic pulmonary fibrosis (IPF), was unsuccessful. The Phase III trial, involving 356 patients, did not meet the primary endpoint of change from baseline in forced vital capacity (FVC) at week 48. The mean decline in FVC was 260 ml in the pamrevlumab arm compared to 330 ml in the placebo arm. The secondary endpoint of time to disease progression was also not met. Consequently, the company will discontinue the ZEPHYRUS-2 phase III study. Earlier in the month, FGEN announced that the phase III LELANTOS-1 placebo-controlled trial of pamrevlumab for the treatment of non-ambulatory patients with DMD on background corticosteroids did not meet the primary endpoint of the Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared with baseline.
Intercept Receives CRL for NASH Treatment
Intercept Pharmaceuticals received a Complete Response Letter (CRL) from the FDA regarding its new drug application (NDA) for obeticholic acid (OCA) in treating pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH). The FDA indicated that the NDA could not be approved in its current form and requires successful completion of the long-term outcomes phase of the REGENERATE study. Intercept will discontinue all NASH-related investments and begin closing out the REGENERATE study, expecting to substantially complete the shutdown by the end of 2023. The company will also cut one-third of its workforce to reduce operating expenses while maintaining its field sales organization to support Ocaliva.
