Agios’ PYRUKYND SNDA Accepted by FDA for Thalassemia; BridgeBio’s BBO-8520 Gets FDA Fast Track for KRASG12C-Mutated Lung Cancer; Nipocalimab Granted FDA Priority Review for Myasthenia Gravis; Rigel’s R289 Receives FDA Orphan Drug Designation for MDS; Rise Therapeutics Gets FDA IND Clearance for R-5780 in Cancer

FDA Accepts Agios’ SNDA for PYRUKYND in Thalassemia Patients

Agios Pharmaceuticals, Inc. announced that the FDA has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) to treat adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The application, assigned a Standard review classification, has a Prescription Drug User Fee Act (PDUFA) target date of September 7, 2025.

“Thalassemia is a rare, lifelong inherited blood disorder that causes chronic anemia and severe complications such as organ damage and stroke, with few effective treatment options available today,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We look forward to collaborating with the FDA during their review process, aiming to provide PYRUKYND, a disease-modifying oral medication, to thalassemia patients, regardless of their genotype or transfusion requirements.”

The sNDA submission is supported by data from the ENERGIZE and ENERGIZE-T Phase 3 trials, which evaluated mitapivat against placebo in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. Results from these pivotal trials were presented at the European Hematology Association 2024 Hybrid Congress and the 66ᵗʰ American Society of Hematology Annual Meeting, highlighting the drug’s potential to transform care for thalassemia patients.

BridgeBio’s BBO-8520 Receives FDA Fast Track Designation for KRASG12C-Mutated Metastatic Lung Cancer

TheRas, Inc., operating as BridgeBio Oncology Therapeutics (BBOT), has announced that the FDA has granted Fast Track designation to its investigational oral therapy, BBO-8520. This designation is for the treatment of adult patients with previously treated KRASG12C-mutated metastatic non-small cell lung cancer (NSCLC). The decision underscores the potential of BBO-8520 to address critical gaps in existing treatment options for this aggressive cancer.

BBO-8520 is designed to inhibit both the “ON” and “OFF” states of KRASG12C, offering optimal target coverage and addressing key resistance mechanisms, such as KRASG12C amplification and receptor tyrosine kinase activation. The therapy has shown substantial tumor growth inhibition in preclinical models, even after resistance to FDA-approved “OFF” inhibitors like sotorasib has developed. This novel approach is the product of a collaboration involving the National Cancer Institute RAS Initiative, Lawrence Livermore National Laboratory, and BBOT.

“Receiving Fast Track designation for BBO-8520 is a significant milestone in our efforts to overcome the limitations of existing therapies for KRASG12C-mutant cancers,” said Yong Ben, M.D., Chief Medical and Development Officer at BBOT. “BBO-8520 represents a first-in-class approach with the potential to address high unmet medical needs and shift the paradigm for cancer treatment. We remain committed to working closely with the FDA to expedite its development, currently under evaluation in a Phase 1 trial (NCT06343402) for KRASG12C NSCLC patients.”

Fast Track designation facilitates the rapid development and review of therapies addressing serious conditions with unmet medical needs, aiming to bring promising treatments to patients more quickly.

Nipocalimab Granted U.S. FDA Priority Review for Generalized Myasthenia Gravis Treatment

Johnson & Johnson announced that the FDA has granted Priority Review designation to its Biologics License Application (BLA) for nipocalimab. The application seeks approval for treating antibody-positive patients (anti-AChR, anti-MuSK, anti-LRP4) with generalized myasthenia gravis (gMG). The designation is supported by findings from the Phase III Vivacity-MG3 study, which demonstrated significant clinical benefits of nipocalimab when combined with standard care.

“We welcome the FDA’s decision to grant Priority Review for the treatment of generalized myasthenia gravis, which underscores the need for additional treatment options in a broad population of people living with gMG,” said Katie Abouzahr, M.D., Vice President of the Autoantibody Portfolio and Maternal Fetal Immunology Disease Area at Johnson & Johnson Innovative Medicine. “We are committed to working closely with the FDA to bring nipocalimab as a potential treatment to patients with gMG, and we extend our gratitude to the participants in the Phase II and III studies. If approved, nipocalimab has the potential to address the needs of individuals with antibody-positive gMG.”

gMG is a rare and chronic autoimmune disorder that affects approximately 700,000 people worldwide, causing symptoms that can range from mild muscle weakness to life-threatening complications. Results from the Phase 3 study showed that nipocalimab, in combination with standard of care, significantly improved MG-ADL scores compared to placebo. Johnson & Johnson has also submitted a Marketing Authorisation Application (MAA) for nipocalimab to the European Medicines Agency (EMA) and continues to explore its potential in other autoimmune conditions, including Sjögren’s disease.

Rigel’s R289 Receives Orphan Drug Designation from the FDA for MDS

Rigel Pharmaceuticals, Inc. announced that the FDA has granted Orphan Drug designation to R289 for the treatment of myelodysplastic syndromes (MDS). R2891 is a potent, selective dual inhibitor of IRAK1 and IRAK4, and is currently being evaluated in a Phase 1b study for its safety, tolerability, pharmacokinetics, and preliminary activity in patients with lower-risk MDS (LR-MDS) who have relapsed or are refractory to prior therapies.

“Receiving Orphan Drug designation for R289 supports the development of this therapeutic candidate for MDS and highlights the significant unmet medical need that exists for these patients,” said Raul Rodriguez, Rigel’s President and CEO. “Orphan Drug and Fast Track designations, coupled with promising initial data from our ongoing Phase Ib study, represent major milestones in advancing R289 as a potential new treatment option for MDS.”

The FDA grants Orphan Drug designation to encourage the development of treatments for rare conditions affecting fewer than 200,000 individuals in the U.S. This designation provides various incentives, including tax credits, exemptions from certain FDA fees, and the potential for seven years of market exclusivity following drug approval. R289 also received Fast Track designation from the FDA for treating previously-treated, transfusion-dependent lower-risk MDS patients.

Rise Therapeutics Gets FDA IND Clearance to Begin Clinical Testing of R-5780 in Cancer

Rise Therapeutics, a biotechnology company focused on developing novel oral immunotherapeutics, announced that the FDA has accepted its investigational new drug (IND) application for R-5780. This marks the company’s fourth clinical program to enter patient testing. The Phase 1 clinical trial will investigate the safety, drug exposure, and clinical activity of R-5780 in patients with cancer. Rise is also conducting ongoing clinical studies for separate products targeting ulcerative colitis, rheumatoid arthritis, and type 1 diabetes.

R-5780 is an oral immune oncology drug candidate designed to enhance the effectiveness of immune checkpoint inhibitors. By engaging gut-regulated immune pathways, R-5780 aims to support a robust anti-tumor T cell response. It represents a pioneering approach in immune oncology, leveraging synthetic biology to direct immune pathways that can potentially expand responses in patients who become refractory to immune checkpoint inhibitors or improve efficacy in initially non-responsive tumors.

Christian Furlan Freguia, Senior Vice President of Research at Rise Therapeutics, commented, “The FDA’s clearance for R-5780 is a testament to the innovation and dedication of our team at Rise Therapeutics. We believe R-5780 has the potential to increase responsiveness in patients and expand treatment options for those with refractory cancers.” The Phase 1 trial (NCT06398418) will enroll up to 33 participants.