LAMA2-CMD is a severe, early-onset form of congenital muscular dystrophy caused by a lack of the LAMA2 protein, which traditional gene therapies have struggled to address due to the protein’s large size. MDL-101, developed using Modalis’ CRISPR-GNDM® technology, is designed to overcome this hurdle by modulating the expression of the LAMA1 gene to compensate for LAMA2 deficiencies. The treatment is poised to be the first gene therapy for LAMA2-CMD that targets the disease’s underlying cause.
“Our mission, ‘Every life deserves attention,’ drives us to innovate for patients who currently have no treatment options,” added Morita. “With this ODD recognition, we’re a step closer to bringing our CRISPR-based therapy to the individuals and families impacted by this devastating condition.”
Pfizer’s RSV Vaccine, ABRYSVO, Gains FDA Approval for High-Risk Adults Aged 18-59
Pfizer Inc. has received FDA approval for ABRYSVO (RSVpreF), the first bivalent RSV vaccine now indicated for adults aged 18-59 at increased risk of RSV-related lower respiratory tract disease. Previously approved for adults over 60 and pregnant individuals in their third trimester, ABRYSVO now has the broadest RSV indication among vaccines for adults, addressing critical protection needs for younger adults with chronic health conditions.
“RSV presents a serious risk for adults with certain chronic conditions,” stated Aamir Malik, Pfizer’s Chief U.S. Commercial Officer and Executive VP. “This approval allows us to better support the high-risk adult population, making ABRYSVO the only RSV vaccine available to adults 18-49 with underlying conditions.”
The FDA’s decision is supported by the Phase III MONeT trial (NCT05842967), which demonstrated ABRYSVO’s safety and immunogenicity in adults with chronic diseases like COPD, diabetes, and heart failure, who are at greater risk of RSV-associated complications. Pfizer plans to publish and present the results at upcoming scientific forums.
With approximately 9.5% of U.S. adults aged 18-49 living with conditions that heighten RSV risk, the expanded indication is poised to provide vital protection to a vulnerable population.
KIND’s AND017 Secures FDA Orphan Drug Designation for Sickle Cell Disease
Kind Pharmaceutical has announced that its investigational drug AND017 has received Orphan Drug Designation (ODD) from the FDA for the treatment of Sickle Cell Disease. This milestone highlights AND017’s potential to provide a novel, effective oral therapy for SCD, a genetic disorder affecting around 120,000 people in the U.S., primarily Black and African American populations.
“The FDA’s ODD for AND017 underscores the urgent need for innovative therapies in SCD,” said Dr. Dong Liu, Founder and CEO of Kind Pharmaceutical. “This designation not only validates our mission to address underserved conditions but also highlights our capabilities in pioneering treatments that could transform lives.”
AND017 is currently in Phase II trials for anemia associated with both non-dialysis-dependent and dialysis-dependent chronic kidney disease (CKD). Results were presented at the American Society of Nephrology’s Kidney Week. Meanwhile, SCD-focused preclinical studies supporting the ODD will be published soon.
“With only limited oral treatments available for SCD, AND017 stands out for its unique mechanism and promising safety profile,” said Professor Gang Huang from UT Health San Antonio, who contributed to AND017’s preclinical studies. “The compelling preclinical data on AND017 suggests significant potential, and I am eager to see how it performs in patients.”
FDA Fast-Tracks HiberCell’s HC-7366 for Relapsed/Refractory AML Treatment
HiberCell, Inc. has announced that the FDA has granted Fast Track designation to HC-7366 for the treatment of adult patients with relapsed or refractory acute myeloid leukemia. This designation will help accelerate the development and review of HC-7366, a novel therapeutic targeting the GCN2 pathway, to address the unmet needs of AML patients.
“We are honored by the FDA’s grant of Fast Track designation for HC-7366,” stated Jonathan Lanfear, President & CEO of HiberCell. “This recognition emphasizes the promise of HC-7366 and highlights the significant need for new treatments in relapsed and refractory AML.”
HC-7366, the first GCN2-activator in clinical testing for AML, is currently in a Phase Ib trial to evaluate its safety and preliminary efficacy in relapsed/refractory AML patients. The study will also establish the recommended Phase II dose (RP2D), with potential for combination and monotherapy expansions based on initial findings.
“With this designation, we are eager to collaborate closely with the FDA to expedite HC-7366’s development and potentially bring a new therapeutic option to AML patients sooner,” Lanfear added.
FDA Approves Iterum’s ORLYNVAH for Treating Uncomplicated UTIs
Iterum Therapeutics has received FDA approval for ORLYNVAH (sulopenem etzadroxil and probenecid), a new oral treatment for uncomplicated urinary tract infections in adult women with limited alternative options. This approval marks the first FDA-approved product for Iterum and introduces the first oral penem in the U.S. market.
“We are thrilled to bring ORLYNVAH™ to patients facing difficult-to-treat uUTIs,” stated Corey Fishman, CEO of Iterum. “This milestone highlights our commitment to addressing antimicrobial resistance and providing an effective alternative for underserved patients.”
The approval is supported by two Phase III trials, SURE 1 and REASSURE, which demonstrated ORLYNVAH’s safety and efficacy. In SURE 1, ORLYNVAH outperformed ciprofloxacin in fluoroquinolone-resistant infections, while in REASSURE, it showed non-inferiority and statistical superiority to Augmentin™ for susceptible infections, with both studies indicating ORLYNVAH™ was generally well tolerated.
“This approval offers a much-needed option for at-risk patients struggling with UTIs,” commented Dr. Marjorie Golden of Yale New Haven Hospital. “ORLYNVAH could be a vital alternative for use in the community based on its impressive clinical profile.”
