As Capricor Therapeutics awaits an FDA decision on its Duchenne muscular dystrophy (DMD) therapy deramiocel, the company has filed a lawsuit against its U.S. commercial partner, NS Pharma, and its parent company, Nippon Shinyaku, alleging failures tied to launch preparations for the treatment.
The lawsuit, filed in the Superior Court of New Jersey, claims that NS Pharma breached its contractual obligations by not sufficiently preparing for the potential commercial rollout of deramiocel. According to Capricor, NS Pharma stopped advancing launch activities after the FDA issued a complete response letter (CRL) for the therapy in July of last year.
Capricor is requesting an injunction that would return U.S. commercialization rights for deramiocel to the company.
“I have spent nearly two decades building Capricor with one goal in mind: making Deramiocel available to treat these boys,” Capricor Chief Executive Officer Linda Marbán, Ph.D., said in a release.
The dispute also centers on pricing terms included in the commercialization agreement between the companies. Capricor stated that the pricing structure negotiated with NS Pharma would prevent patients covered by Medicare, Medicaid, or private insurance from obtaining access to the therapy. The company said NS Pharma had declined to make changes to address the issue.
In the complaint, Capricor stated that neither party recognized at the time the agreement was finalized that the pricing arrangement would not be workable. The company argued that the agreed structure would make it financially impractical to distribute deramiocel to Medicaid beneficiaries and privately insured patients because the selling price could fall below manufacturing and distribution costs.
NS Pharma and Nippon Shinyaku disputed Capricor’s allegations in a response to the lawsuit. The companies stated that they had acted appropriately and in good faith to help ensure the therapy could reach DMD patients if approved. They also said they remained open to discussions with Capricor regarding the value of CAP-1002, also known as deramiocel, while maintaining that the claims in the lawsuit lacked merit.
The allogeneic cardiac-derived cell therapy received a CRL from the FDA in 2025, a decision that Capricor said was a surprise because the company believed it had previously agreed with the agency that its phase 2 trial would be sufficient to support approval.
Several months later, Capricor announced that the FDA had resumed review of the application after lifting the CRL. The agency assigned an August 22 Prescription Drug User Fee Act (PDUFA) target date for a decision on the treatment.
The FDA’s change in position on deramiocel came during a period under former Center for Biologics Evaluation and Research chief Vinay Prasad, M.D., who oversaw multiple rejections of rare disease therapies that biotech companies said had come as surprises. The period also included a public dispute with gene therapy developer uniQure that the original report said may have contributed to Prasad’s departure.
Since Capricor announced the lawsuit after markets closed Thursday, the company’s shares declined 13%, while shares of Nippon Shinyaku fell 15%.
Capricor Therapeutics has initiated legal action against NS Pharma regarding alleged launch preparations tied to Deramiocel before an official FDA decision. The dispute places Capricor at the center of a growing legal and regulatory battle within the Duchenne muscular dystrophy (DMD) treatment space.
Background of the Capricor Lawsuit
Capricor alleges that NS Pharma engaged in activities related to commercialization and market preparation for Deramiocel before receiving final regulatory clearance from the U.S. Food and Drug Administration.
Duchenne muscular dystrophy (DMD) is one of the most severe genetic neuromuscular disorders, primarily affecting young boys. The disease is caused by mutations in the dystrophin gene, leading to progressive muscle weakness, loss of mobility, respiratory complications, and cardiac issues over time.
Despite advances in supportive care, treatment options remain limited, making the development of innovative therapies a high priority for researchers, patient organizations, and healthcare providers worldwide.
Importance of Cell Therapy in Rare Diseases
Cell-based therapies have emerged as a promising area of research for neuromuscular disorders. Unlike traditional treatments that mainly manage symptoms, regenerative approaches aim to repair damaged tissues, reduce inflammation, and potentially slow disease progression at a biological level.
