This global trial is designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with sickle cell disease aged 12 years or older. About Mitapivat in Sickle Cell Disease Sickle cell disease is a rare, inherited blood disorder in which abnormal hemoglobin ... This global trial is designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with sickle cell disease aged 12 years or older. About Mitapivat in Sickle Cell Disease Sickle cell disease is a rare, inherited blood disorder in which abnormal hemoglobin causes red blood cells to become rigid and sickle-shaped, leading to chronic hemolysis (red blood cell destruction) that contributes to severe anemia and progressive, life-threatening complications, including damage to the lungs, kidneys, and heart. P-gp Substrates: Monitor patients for adverse reactions of the substrates with narrow therapeutic index. Hepatic Impairment Avoid use of AQVESME in patients with cirrhosis (Child-Pugh Class A, B, or C). Please see full Prescribing Information for AQVESME, including Boxed Warning. About Agios: Fueled by Connections to Transform Rare Diseases™ At Agios, our vision is to redefine the future of rare disease treatment. On October 19, 2026, Agios submitted its response, which included approximately nine months of updated safety data through October 9, 2026, from the ongoing open-label extension periods of the RISE UP Phase 2 and Phase 3 trials. With this additional follow-up, there continue to be no cases of hepatocellular injury (HCI) among patients with sickle cell disease treated with mitapivat that show a pattern and severity similar to those observed in the clinical trials for mitapivat in adults with thalassemia. About the RISE UP Phase 3 Trial The global RISE UP Phase 3 trial (NCT05031780) was designed to evaluate the efficacy and safety of mitapivat in patients with sickle cell disease aged 16 years or older, representative of the global population. The trial design encompassed a 52-week, double-blind, randomized, placebo-controlled period, in which 207 participants were randomized 2:1 to receive oral mitapivat (100 mg) twice daily (n=138) or matched-placebo (n=69), followed by an open-label extension (OLE) period, during which all participants receive mitapivat. To evaluate the effect of mitapivat o

Early modeling put sickle cell gene therapy coverage near 11 cents per member per month. Cell and gene therapies (CGTs) are spreading beyond oncology and rare diseases, but the way health plans pay for them has not kept pace. On portability, he said, “all of these costs for gene therapies are up front, but the savings that might come from these potentially curative medical miracles actually don’t accrue for years afterwards,” and 1 in 5 Americans leave their health plan each year. Regarding administration, he said over half of patients with sickle cell disease do not live in the same metro area as an authorized treatment center, and manufacturing capacity may be as low as 1 to 2 patients per year. States must publish coverage criteria, pay separately for the therapy, reimburse at least the acquisition cost, and contract with a qualified treatment center, including out of state, if needed. Singh said CMS announced on October 27 a request for applications to add other therapies.7 The model is “a great step forward in terms of helping address some of the challenges with financing CGTs. But I wouldn’t say that it addresses everything.” · During the audience question-and-answer period, Singh said sickle cell disease’s lifetime direct medical costs may run $2 million to $6 million,8 so “lifetime costs of care are relevant when putting the price of gene therapy in context.” Asked whether manufacturing efficiencies could cut costs, he said, “Whether that yields sort of list price reductions, I’m not so sure. rare disease leading. Immunology, neurology, sensory, and cardiovascular indications are expanding, and “the need for health plans to have financing and reimbursement pathways grows as the number of patients who are potentially treated by these gene therapies also grows.”

Cardiologists who study gene therapy explain its promise, risks and the safeguards needed for responsible use. Early successes in gene therapy have focused on these rare disorders, such as spinal muscular atrophy and sickle cell disease. Gene therapy is no longer the future of medicine — it’s reality. Treatments that replace, silence or edit genes are already changing clinical care. There are more than 6,000 rare genetic disorders but only a fraction currently have treatable targets. Current gene therapies target somatic cells, meaning any genetic changes are not intended to be passed to future generations. Murthy: For rare, severe diseases, the case can be relatively straightforward: The unmet need is high, and patients may have few or no effective alternatives. Some currently available gene therapies have list prices exceeding $1 million per patient, with several priced substantially higher. While a one-time therapy may make sense for a rare, life-threatening disease, similar pricing models would be difficult to sustain for conditions affecting millions of people.

Mehmet Oz, who leads the Centers ... for rare diseases. Oz was joined by Cleveland Clinic CEO Dr. Tomislav Mihaljevic, acting FDA Commissioner Kyle Diamantas and CMS Deputy Administrator Abe Sutton, according to Ideastream. The venue was no accident: Cleveland Clinic has led research initiatives using gene therapies for inherited blood disorders including sickle cell anemia and ... Mehmet Oz, who leads the Centers for Medicare & Medicaid Services, visited Cleveland to explain federal guidelines easing gene therapy approval for rare diseases. Oz was joined by Cleveland Clinic CEO Dr. Tomislav Mihaljevic, acting FDA Commissioner Kyle Diamantas and CMS Deputy Administrator Abe Sutton, according to Ideastream. The venue was no accident: Cleveland Clinic has led research initiatives using gene therapies for inherited blood disorders including sickle cell anemia and thalassemia, putting it at the center of a national conversation about how to get these treatments to patients faster and cheaper. Cleveland Clinic just became ground zero for a federal plan to speed up pricey gene therapies. Federal officials visited Cleveland Clinic to outline plans speeding gene-therapy approvals and reshaping Medicaid pricing for rare diseases. Thirty-three states, the District of Columbia and Puerto Rico have signed agreements to participate in the Cell and Gene Therapy Access Model, according to the Centers for Medicare & Medicaid Services, and the program could run for as long as 11 years. Oz said the initiative would give states with relatively few rare-disease patients access to better drug-maker pricing, and that federal guidelines would speed gene-therapy approvals while making the resulting care easier for Medicaid patients to access, per the account relayed by Ideastream.

CMS is planning to open the Cell and Gene Therapy Access model to allow centralized outcomes-based contracting for gene therapies in the Medicaid program, but while the current sickle cell disease program is growing rapidly, the success may be difficult to emulate. CMS is planning to open the Cell and Gene Therapy Access model to allow centralized outcomes-based contracting for gene therapies in the Medicaid program, but while the current sickle cell disease program is growing rapidly, the success may be difficult to emulate. CMMI is preparing for another sickle cell treatment to enter the Cell And Gene Therapy Access Model. NervGen's RESTORE trial, which dosed its first patient Oct. 12, is a rare pivotal spinal cord injury study built on the patient-informed functional primary endpoint GRASSP as sponsors look beyond motor-based outcomes. ... The FDA's drugs and biologics center directors said lessons from the rare disease “mindset shift” could be leveraged for chronic condition drug development, especially where patient subsets can be identified. 27 that the Cell and Gene Therapy Access Model will move to include non-sickle cell treatments. The US Centers for Medicare and Medicaid Services will soon open its Cell and Gene Therapy Access Model to non-sickle... Derrick Gingery and the team bring you a weekly "Drug Fix". Share current article via EmailShare current article via TwitterShare current article via LinkedIn ... Stemirac set to make first successful transition from conditional to full approval for a cell therapy in Japan, but faces commercial challenges.