Opdivo Qvantig provides a quicker administration alternative, delivering immunotherapy in just three to five minutes compared to the 30-minute infusion time for IV Opdivo. The trial results showed a slightly higher overall response rate (ORR) of 24% with Opdivo Qvantig compared to 18% with IV Opdivo, along with non-inferior pharmacokinetics and a comparable safety profile. This new method offers greater flexibility, allowing treatment closer to home and reducing preparation steps for providers.
“At Bristol Myers Squibb, we remain dedicated to improving every aspect of the patient care journey,” stated Adam Lenkowsky, executive vice president and chief commercialization officer. “This approval adds another layer of flexibility and convenience to Opdivo’s proven track record in immunotherapy, reinforcing our commitment to patient-centric innovation and care.”FDA Accepts Nuvation Bio’s Taletrectinib NDA for Advanced ROS1-Positive Non-Small Cell Lung Cancer
FDA Accepts Nuvation Bio’s Taletrectinib NDA for Advanced ROS1-Positive NSCLC
Nuvation Bio Inc. has announced the FDA’s acceptance of its New Drug Application (NDA) for taletrectinib, a next-generation ROS1 tyrosine kinase inhibitor (TKI). The investigational therapy targets advanced ROS1-positive non-small cell lung cancer (NSCLC), regardless of prior treatment. The FDA has granted Priority Review to the application, setting a Prescription Drug User Fee Act (PDUFA) goal date of June 23, 2025. This designation highlights the potential of taletrectinib to address the significant unmet needs for patients with ROS1-positive NSCLC. Notably, taletrectinib has also received Orphan Drug Designation and Breakthrough Therapy Designation, making it the only ROS1 TKI in development with such recognition.
“We are thrilled to reach this important milestone for taletrectinib, a significant step forward for people living with ROS1-positive NSCLC who urgently need new treatment options,” said Dr. David Hung, Founder, President, and CEO of Nuvation Bio. “With data from over 300 patients—the largest ROS1-positive NSCLC dataset to date supporting an original NDA—taletrectinib has demonstrated the potential to deliver durable and meaningful benefits.”
The NDA submission is supported by pooled results from the pivotal Phase II TRUST-I and TRUST-II studies, previously presented at the European Society of Medical Oncology (ESMO) Congress in September 2024. These studies highlighted taletrectinib’s potential to offer significant clinical benefits for patients, marking an advancement in the treatment of advanced ROS1-positive NSCLC.
Dr. Hung added, “Since acquiring AnHeart Therapeutics earlier this year, including taletrectinib, we have executed on our plan to advance taletrectinib toward a full U.S. regulatory approval. The FDA’s Priority Review reflects the strength of our clinical data and the promise taletrectinib holds for patients. As we prepare for a launch as early as mid-2025, we’re taking critical steps to establish Nuvation Bio as a commercial oncology organization, reinforcing our commitment to bringing innovative therapies to patients who need them most.”
HKBU’s Aptamer for X-Linked Hypophosphatemia Receives Orphan and Rare Pediatric Disease Designations from FDA
A collaboration between Hong Kong Baptist University (HKBU) and Shanghai Sixth People’s Hospital has resulted in the development of a novel therapeutic aptamer for treating X-linked hypophosphatemia (XLH), a rare genetic bone disease. Originally designed to treat osteogenesis imperfecta, the aptamer has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the FDA, accelerating its path toward clinical use and market availability.
XLH, caused by mutations in the PHEX gene, leads to poor bone mineralization, resulting in symptoms like growth retardation, limb deformities, and osteomalacia. Existing treatments face limitations, particularly due to associated cardiovascular risks. The newly developed aptamer, Apc001, targets the “loop3 domain” of the sclerostin protein, enhancing bone formation while preserving cardiovascular safety.
Animal studies have shown promising results, including increased blood phosphorus levels and improved bone strength in XLH-affected models. With pilot-scale production completed and preclinical toxicological assessments underway, Apc001 is scheduled to begin clinical trials in both Mainland China and the U.S., offering hope for more effective and safer treatment options for XLH patients.
FDA Grants Orphan Drug Status to Sapience Therapeutics’ ST316 for Familial Adenomatous Polyposis
Sapience Therapeutics, Inc., announced that the FDA has awarded Orphan Drug Designation to ST316 for the treatment of familial adenomatous polyposis (FAP). FAP is a rare pre-malignant genetic condition that causes the growth of numerous polyps in the colon, often starting in adolescence. Without surgical intervention, the condition invariably progresses to colorectal cancer (CRC) by age 40, with no approved therapies currently available.
ST316 is a first-in-class therapy designed to target the Wnt/β-catenin signaling pathway, which is central to the development of FAP and over 80% of CRCs. Currently in a Phase II trial for CRC, ST316 inhibits β-catenin and its co-activator, BCL9, aiming to prevent the progression of FAP to malignancy and address a significant unmet medical need.
“We are thrilled that the FDA has granted Orphan Drug Designation to ST316 for the treatment of FAP,” said Dr. Abi Vainstein-Haras, Sapience’s Chief Medical Officer. “This milestone underscores the potential of ST316 to intervene in both pre-malignant and malignant diseases driven by genetic alterations in the Wnt/β-catenin signaling pathway. Patients with FAP currently have no treatment options other than surgery and intensive monitoring to manage their disease.”
Dr. Vainstein-Haras added, “The FDA’s recognition of ST316 highlights the importance of addressing the Wnt/β-catenin pathway in both FAP and CRC. We are committed to advancing the development of ST316 and providing a meaningful therapeutic option for patients with FAP, who face a high risk of colorectal cancer, the second-leading cause of cancer death in the United States.”
Orphan Drug Designation offers several benefits, including eligibility for federal grants, tax credits for clinical research, exemption from certain FDA fees, and seven years of market exclusivity upon approval. These incentives aim to accelerate the development of treatments for rare diseases, offering hope to patients with limited options.
Precigen Submits BLA for PRGN-2012 in Recurrent Respiratory Papillomatosis, Requests FDA Priority Review
Precigen, Inc. has finalized the rolling submission of a Biologics License Application (BLA) to the FDA for PRGN-2012, a groundbreaking AdenoVerse® gene therapy. This investigational treatment targets adult patients with recurrent respiratory papillomatosis (RRP), a rare and life-altering disease caused by HPV 6 or HPV 11. The BLA is now under a 60-day FDA review, during which the agency will decide on acceptance and determine a potential Prescription Drug User Fee Act (PDUFA) action date. If granted priority review, the timeline for evaluation will be reduced to six months.
PRGN-2012 holds significant potential as the first FDA-approved therapeutic for RRP, a condition marked by persistent, surgery-requiring papillomas in the respiratory tract. Current treatments involve repetitive surgeries that fail to address the root cause and significantly impact patient quality of life. Data from a pivotal Phase I/II study, presented at the 2024 ASCO Annual Meeting, demonstrated the safety and efficacy of PRGN-2012. Primary endpoints, including safety and complete response rates (patients requiring no surgeries within 12 months of treatment), were successfully met.
“The impact of this debilitating disease on patients, families, and their caregivers has been overlooked for more than half a century,” said Dr. Helen Sabzevari, President and CEO of Precigen. “There is currently no approved therapy for RRP patients, and the submission of our BLA is an extremely important step in bringing the first therapy to fight this devastating disease.”
Dr. Sabzevari further noted, “We look forward to working closely with the FDA on next steps and are excited by the potential to bring PRGN-2012 to RRP patients as quickly as possible. With our recent financial strategies enhancing our cash runway into 2026, we are well-positioned for a potential commercial launch in the second half of 2025.”
This milestone submission reflects Precigen’s commitment to addressing critical unmet medical needs through innovative gene and cell therapies.
