Dr. Irene Wang, President and Chief Scientific Officer of Lin BioScience, highlighted the positive safety profile of LBS-007, stating, “During the Phase I dose escalation, we have not observed any Definitely or Probably Related Adverse Effects in all patients who have received low to high doses, demonstrating the safety profile is very tolerable and promising.”
FDA Reviews Alnylam’s sNDA for Expanded AMVUTTRA Use
Alnylam Pharmaceuticals has announced that the FDA has accepted its supplemental new drug application (sNDA) for AMVUTTRA (vutrisiran) to expand its label for the treatment of transthyretin amyloidosis with cardiomyopathy. AMVUTTRA, an RNA interference (RNAi) therapeutic, is currently approved for treating adult patients with polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis in the U.S. and in the EU for hATTR amyloidosis in adults with stage 1 or 2 polyneuropathy.
The FDA has accepted the sNDA for review under the Priority Review pathway, which shortens the review period to four months. A final decision on the application is expected by March 23, 2025. Notably, the FDA has indicated that it will not convene an advisory committee meeting for this application, streamlining the approval process. If approved, AMVUTTRA will become the first therapy in the U.S. to treat both the polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.
The application for expanding AMVUTTRA’s indication is supported by the positive results from Alnylam’s pivotal Phase III HELIOS-B study, which met all primary and secondary endpoints, demonstrating statistical significance in both the overall and monotherapy populations. These promising results have further bolstered the potential of vutrisiran in treating ATTR-CM.
Alnylam’s progress has also been reflected in its stock performance, with shares gaining 31.1% year-to-date, outperforming the broader industry, which has seen a 9% decline. This milestone marks a significant step forward in the company’s commitment to providing innovative treatments for rare and serious diseases like ATTR amyloidosis.
FDA Approves AOP Health’s RAPIBLYK (Landiolol) for Atrial Fibrillation and Flutter in Critical Care
AOP Health, based in Vienna, Austria, announced that the FDA had granted approval for RAPIBLYK (landiolol) in the critical care setting for the treatment of supraventricular tachycardia, specifically atrial fibrillation, and atrial flutter. This approval provides U.S. patients with a new therapeutic option for rapidly managing heart rate in intensive care units. RAPIBLYK (landiolol) has shown significant promise in clinical studies, demonstrating effective heart rate management with minimal impact on blood pressure.
The approval is based on data from five randomized, double-blind, placebo-controlled clinical trials, involving 317 adults with supraventricular tachycardia. The studies revealed that RAPIBLYK (landiolol) was effective in reducing the heart rate by 40-90% within about 10 minutes, compared to 0-11% in the placebo group. Heart rate reduction was defined as a more than 20% decrease in heart rate, a heart rate of less than 100 bpm, or at least intermittent cessation of the arrhythmia. While some adverse events were reported in the landiolol group (9.9%), these were much fewer than in the placebo group (1%).
This approval marks an important milestone for AOP Health, as it expands its critical care offerings to the U.S. market. Dr. Martin Steinhart, CEO of AOP Health, expressed the significance of the approval, stating, “RAPIBLYK approval in the U.S. represents an important milestone for patients experiencing supraventricular tachycardia, including atrial fibrillation and atrial flutter, who need rapid and short-term heart rate reduction.” He added, “We are delighted that this therapeutic option, already available in Europe, can now help U.S. patients as well.”
AOP Health continues to focus on providing solutions for rare diseases and critical care, and this approval strengthens the company’s mission to make a meaningful impact on patient outcomes, particularly in the treatment of severe heart conditions like atrial fibrillation and atrial flutter.
Applied Therapeutics Receives Complete Response Letter from FDA for Govorestat NDA for Classic Galactosemia
Applied Therapeutics, Inc. announced that the FDA has issued a Complete Response Letter (CRL) for its New Drug Application (NDA) for govorestat. Govorestat is a novel, CNS-penetrant aldose reductase inhibitor (ARI) being developed for the treatment of Classic Galactosemia. The FDA’s decision was based on deficiencies identified in the clinical application, meaning the NDA cannot be approved in its current form.
The company is reviewing the FDA’s feedback and plans to request a meeting with the agency to discuss the next steps, including potential resubmission of the NDA or an appeal. Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics, expressed disappointment in the decision, stating, “Our strong commitment to the Galactosemia community is rooted in our belief that govorestat has the potential to change the lives of patients with Galactosemia, as evidenced by the breadth of efficacy and safety data.”
Despite the setback, govorestat has shown promising results in clinical trials, demonstrating rapid and sustained reductions in galactitol levels, along with clinical benefits in children with Galactosemia aged 2-17. In the Phase III ACTION-Galactosemia Kids study, govorestat improved daily activities, cognition, behavior, and fine motor skills, while also reducing tremors. Additionally, govorestat has displayed a favorable safety profile, further supporting its potential as a therapeutic option for this underserved patient population.
Beyond Classic Galactosemia, govorestat is also being developed for Sorbitol Dehydrogenase (SORD) Deficiency, a rare neuromuscular disease. The company anticipates submitting an NDA for SORD in early 2025. Importantly, the development of govorestat for SORD is independent of the ongoing review for Classic Galactosemia. Applied Therapeutics remains dedicated to addressing the high unmet medical need for patients with these rare conditions.
Rigel Announces FDA Fast Track Designation for R289 in Lower-Risk MDS
Rigel Pharmaceuticals, Inc. announced that the FDA has granted Fast Track designation to R289 for the treatment of patients with previously treated, transfusion-dependent lower-risk myelodysplastic syndrome (LR-MDS). R289, a potent and selective dual inhibitor of IRAK1 and IRAK4, is currently being evaluated in an ongoing Phase Ib study. The study is focused on assessing the safety, tolerability, pharmacokinetics, and preliminary activity of R289 in LR-MDS patients who have relapsed or are refractory to prior treatments.
Raul Rodriguez, Rigel’s president and CEO, expressed his enthusiasm for the Fast Track designation, stating, “We are pleased that R289 has been granted Fast Track designation, which underscores the significant unmet need for patients with transfusion-dependent lower-risk MDS. By targeting inflammatory signaling, we believe that R289 has the potential to meaningfully improve the lives of those living with this disease.”
Dr. Lisa Rojkjaer, Rigel’s chief medical officer, highlighted the importance of the designation for the primarily elderly LR-MDS patient population, who face progressive cytopenias, particularly anemia. “This designation is based on initial data from the ongoing Phase 1b study and highlights the potential of R289 to be a new therapeutic option for these patients,” she said. “We look forward to working closely with the FDA to advance the clinical development of R289.”
