Supported by the CAHtalyst Phase III clinical trials, the largest-ever program in CAH, CRENESSITY demonstrated significant reductions in adrenal androgens and glucocorticoid doses in both pediatric and adult patients. In pediatric patients, CRENESSITY reduced androstenedione levels approximately four times more than placebo, while enabling meaningful glucocorticoid dose reductions. Adults taking CRENESSITY achieved up to an eight-fold greater reduction in androstenedione levels compared to placebo, with 63% reaching glucocorticoid doses within the physiologic range.
“Patients with CAH struggle to balance symptoms and the side effects of high-dose steroids, impacting their quality of life,” said Dina Matos, Executive Director of CARES Foundation. “CRENESSITY offers new hope, reducing androgen excess and steroid dependence.”
CRENESSITY is expected to launch within a week through PANTHERx Rare specialty pharmacy, with comprehensive patient support through Neurocrine Access Support. Most patients will pay $10 or less per month for the medication.
Checkpoint Therapeutics Announces FDA Approval of UNLOXCYT
Checkpoint Therapeutics, Inc. announced that the FDA has approved UNLOXCYT™ (cosibelimab-ipdl) for adults with metastatic cutaneous squamous cell carcinoma or locally advanced cSCC who are not candidates for curative surgery or radiation. UNLOXCYT is the first FDA-approved PD-L1–blocking antibody for this indication, offering a differentiated treatment option with its ability to induce antibody-dependent cell-mediated cytotoxicity (ADCC).
“Today’s approval of UNLOXCYT marks a significant milestone for both Checkpoint and patients with advanced cSCC,” said James Oliviero, CEO of Checkpoint Therapeutics. “This positions us to compete in a U.S. market exceeding $1 billion annually, providing a unique therapy that binds to PD-L1 to restore anti-tumor immune response.”
UNLOXCYT’s approval is supported by objective response rates and durable responses observed in the CK-301-101 trial. “Patients with advanced cSCC, particularly those with comorbidities, face limited treatment options,” said Dr. Emily Ruiz of Harvard Medical School. “UNLOXCYT’s safety profile and efficacy represent a promising new immunotherapy for oncologists.”
Checkpoint is preparing for the commercial launch of UNLOXCYT, with the recommended dosage being 1,200 mg via intravenous infusion every three weeks. Mr. Oliviero expressed gratitude to patients, physicians, and the FDA for their role in achieving this critical approval.
MAIA Biotechnology Receives FDA Rare Pediatric Disease Designation for THIO
MAIA Biotechnology, Inc. announced that the FDA has granted Rare Pediatric Disease Designation to THIO for the treatment of pediatric-type diffuse high-grade gliomas, one of the most treatment-resistant childhood cancers. THIO, a novel anti-cancer agent, activates the immune system while overcoming tumor immunosuppression, presenting a promising therapeutic option.
“THIO has demonstrated positive results in treating difficult cancers, including pediatric high-grade gliomas,” said Vlad Vitoc, M.D., Chairman and CEO of MAIA. “This designation strengthens our commitment to advancing research for this devastating childhood disease.”
K. Robinson Lewis, MAIA’s Vice President and Head of Regulatory, emphasized the value of this designation, as it makes MAIA eligible for a priority review voucher (PRV) upon FDA approval. PRVs, which can be redeemed or sold, have historically commanded valuations averaging $100 million.
THIO has previously shown strong efficacy in diffuse intrinsic pontine glioma (DIPG) in preclinical studies, particularly when combined with ionizing radiation. The promising results were presented at the 2024 AACR Annual Meeting. In addition to PDHGG, THIO holds orphan drug designations for hepatocellular carcinoma, small cell lung cancer, and glioblastoma.
JEMPERLI gets FDA Breakthrough Therapy Designation for Rectal Cancer
GSK plc announced that the FDA has granted Breakthrough Therapy Designation to JEMPERLI (dostarlimab) for the treatment of patients with locally advanced mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) rectal cancer. This designation accelerates the development of therapies with significant potential to improve treatment outcomes for serious conditions. It follows the Fast Track designation for the same indication in January 2023.
The designation is based on results from a Phase II trial conducted in collaboration with Memorial Sloan Kettering Cancer Center, where 100% of 42 patients completing dostarlimab treatment achieved a clinical complete response (cCR) with no evidence of tumors. “Today’s designation, based on this unprecedented response rate, supports a path to change the treatment paradigm for patients facing significant long-term quality-of-life effects,” said Hesham Abdullah, SVP, Global Head Oncology, R&D, GSK.
Current standard treatment involves chemotherapy, radiation, and surgery, which can result in long-term complications such as bowel dysfunction, infertility, and secondary cancers. GSK’s ongoing AZUR-1 registrational trial aims to confirm the promising results of dostarlimab as a potential frontline therapy for this patient population, offering new hope for improved outcomes and quality of life.
Galderma Announces FDA Approval of NEMLUVIO for Atopic Dermatitis
Galderma announced that the FDA has approved NEMLUVIO (nemolizumab) for the treatment of patients aged 12 and older with moderate-to-severe atopic dermatitis, in combination with topical corticosteroids (TCS) and/or calcineurin inhibitors (TCI). This approval follows NEMLUVIO’s earlier FDA approval in August 2024 for adults with prurigo nodularis, further expanding its therapeutic reach.
“NEMLUVIO is an important and effective new treatment option for patients with atopic dermatitis, where unmet needs remain,” said Flemming Ørnskov, M.D., MPH, CEO of Galderma. “This FDA approval marks a significant milestone, underscoring our commitment to delivering innovative solutions to patients and accelerating the growth of our Therapeutic Dermatology Business.”
The approval is based on positive results from the Phase III ARCADIA clinical trials, involving 1,728 patients. The data showed that NEMLUVIO, administered subcutaneously every four weeks, achieved significant improvements in skin clearance and a 75% reduction in the Eczema Area and Severity Index (EASI) at 16 weeks, compared to placebo. NEMLUVIO also delivered rapid itch relief as early as Week 1 and significantly improved sleep disturbance.
NEMLUVIO was well-tolerated, with a safety profile consistent across treatment and placebo groups. In addition to the FDA approval, the European Medicines Agency’s (EMA) CHMP has issued a positive opinion recommending NEMLUVIO for atopic dermatitis and prurigo nodularis in Europe. Galderma anticipates peak sales for NEMLUVIO to exceed $2 billion, approaching “blockbuster” status by 2027.
Galderma continues to seek approvals globally, with regulatory reviews underway in Australia, Canada, Brazil, South Korea, and other key markets.
