Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths

Sarepta Therapeutics, Inc. has voluntarily and temporarily paused all shipments of ELEVIDYS (delandistrogene moxeparvovec) in the U.S., effective July 22, 2025, following engagement with the FDA on safety labeling updates. The decision comes amid the ongoing label supplement process and post-marketing safety discussions after adverse events were reported in patients with Duchenne muscular dystrophy (DMD).

“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,” said Doug Ingram, President and CEO of Sarepta. “Maintaining a positive and productive working relationship with the FDA is critical as we work collaboratively to complete the safety review process.”

ELEVIDYS, Sarepta’s single-dose gene therapy, was granted accelerated approval for use in ambulatory and non-ambulatory DMD patients aged four years and older with confirmed mutations in the DMD gene. Continued approval for non-ambulatory patients is contingent on confirmatory trial results demonstrating clinical benefit. The therapy is contraindicated in patients with deletions in exon 8 and/or exon 9 due to risks of immune-mediated myositis and other severe reactions.

Sarepta confirmed its continued commitment to transparency and patient safety. “This pause will allow us to respond to the FDA’s requests and complete the necessary label supplement process without compromising trust or safety,” Ingram added. The company intends to provide timely updates to the DMD community as more information becomes available.

ImCheck’s ICT01 Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia

ImCheck Therapeutics announced that the FDA has granted Orphan Drug Designation (ODD) to its lead candidate, ICT01, for the treatment of acute myeloid leukemia (AML). The designation follows compelling results from the Phase I/II EVICTION study presented at the 2025 ASCO Annual Meeting.

“Receiving FDA orphan drug designation for ICT01 is a significant recognition of ICT01’s innovative therapeutic potential to meet the urgent unmet medical needs of AML patients,” said Dr. Stephan Braun, Chief Medical Officer at ImCheck Therapeutics. “This milestone reinforces our confidence that ICT01 will become the first immunotherapy for AML patients.”

ICT01, a humanized anti-BTN3A monoclonal antibody, selectively activates γ9δ2 T cells and is being evaluated in combination with azacitidine and venetoclax in newly diagnosed AML patients who are unfit for intensive chemotherapy. The treatment showed unprecedented remission rates and a positive overall survival signal across genetically diverse subgroups, with a safety profile consistent with standard hematological toxicities.

“Orphan drug designation is a catalyst,” added Pierre d’Epenoux, CEO of ImCheck. “It validates our regulatory strategy, de-risks development, and accelerates our efforts to position ICT01 as a transformative therapy in AML and potentially other solid tumors.”

RedHill Biopharma Gets Positive FDA Feedback on RHB-204 for Crohn’s Disease Approval Pathway

RedHill Biopharma announced today that the FDA has provided constructive guidance for advancing its lead candidate, RHB-204, into a novel phase II study for Crohn’s disease (CD). The trial is expected to be the first to enroll only Mycobacterium avium subspecies paratuberculosis (MAP)-positive CD patients—potentially validating MAP as a root cause of the disease and positioning RHB-204 as a paradigm-shifting therapy targeting both etiology and symptoms.

“This FDA feedback supports a pioneering approach to Crohn’s disease, and we’re excited to lead the first study specifically designed to assess treatment efficacy in MAP-positive patients,” said Dr. Reza Fathi, RedHill’s Senior VP of R&D. “We believe RHB-204 could offer a safer, more effective oral alternative to current therapies, especially for patients unresponsive to biologics.”

The phase II trial will utilize cutting-edge MAP detection diagnostics, addressing a key historical barrier in MAP-related research. RedHill has established collaborations with two academic institutions to implement these rapid tests. The trial will focus on mucosal and clinical remission as primary endpoints, in alignment with FDA guidance and supported by data from the earlier phase III RHB-104 study, which showed a 64% improvement in efficacy over standard of care.

“RHB-204 builds on the success of RHB-104 and represents a major step forward in treating CD with a novel, oral immunomodulatory strategy,” said Guy Goldberg, CEO of RedHill. “With strong IP protection through 2041 and non-dilutive funding pathways under discussion, we’re well-positioned to deliver a transformational therapy in a market projected to exceed $19 billion by 2033.”

FDA Grants Fast Track Designation to ProMIS Neurosciences’ PMN310 for Alzheimer’s Disease

ProMIS Neurosciences announced that the FDA has granted Fast Track designation to its lead antibody therapy, PMN310, currently being developed for Alzheimer’s disease (AD). The designation is intended to expedite the development of drugs that target serious conditions with unmet medical needs and will allow ProMIS to benefit from enhanced regulatory guidance and a streamlined path toward potential approval.

“This is a pivotal moment for ProMIS and the Alzheimer’s community,” said Neil Warma, President and CEO of ProMIS Neurosciences. “Receiving Fast Track designation not only underscores the potential of PMN310 to address a critical unmet need but also provides valuable opportunities for regulatory insight as we advance toward key clinical milestones.”

PMN310 is designed to selectively target toxic misfolded forms of amyloid-beta, which are believed to drive AD pathology while sparing normal forms that may play protective roles. This mechanism may reduce the incidence of amyloid-related imaging abnormalities (ARIA)—serious side effects such as brain swelling and bleeding observed with other AD therapies—while delivering stronger efficacy.

The ongoing PRECISE-AD phase Ib trial is assessing PMN310 in early-stage Alzheimer’s patients, focusing on safety, tolerability, pharmacokinetics, and disease-relevant biomarkers. Interim six-month biomarker and safety data are expected in Q2 2026, with final results anticipated in Q4 2026.

Oruka Therapeutics Receives IND Clearance for Phase IIa Trial of ORKA-001 in Psoriasis

Oruka Therapeutics has received FDA clearance to initiate EVERLAST-A, a randomized, double-blind, placebo-controlled Phase IIa trial evaluating ORKA-001, a long-acting anti-IL-23p19 antibody, in moderate-to-severe psoriasis. The trial will enroll approximately 80 patients, randomized 3:1 to receive ORKA-001 or placebo, with the primary endpoint being PASI 100 at Week 16. Initial efficacy and response duration data are expected in the second half of 2026.

“We’re thrilled by this rapid progress into Phase II development, and very excited to start our first psoriasis study,” said Lawrence Klein, PhD, CEO of Oruka. “We think that ORKA-001 can redefine the standard of care in this important disease with potential for once-per-year dosing, off-treatment remissions, and higher rates of disease clearance.”

EVERLAST-A also includes an extended dosing component: patients who achieve PASI 100 by Week 28 will be randomized 2:1 into a no-dose arm or biannual dosing arm to evaluate the durability of remission. Additional design details and interim Phase I data will be presented at the upcoming EADV Congress in September.

Dr. Bruce Strober, lead Principal Investigator of the EVERLAST-A study, commented, “I’m excited about EVERLAST-A and the potential that ORKA-001 could enable long dosing intervals and greater efficacy than current IL-23 inhibiting medications. These improvements could be a significant step forward for both patients and the field.”