Cell therapy weekly: Representatives reject ‘right to try’ and Duchenne trial placed on hold
This week: US House of Representatives votes against tabled ‘right to try’ bill and a new treatment for osteoarthritis receives RMAT designation.
The news highlights:
US House of Representatives rejects ‘right to try’ bill
Micronized amniotic tissue granted RMAT for the treatment of osteoarthritis
Phase I/II Duchenne muscular dystrophy trial placed on clinical hold
US House of Representatives rejects ‘right to try’ bill
Proposed legislation that would cut the FDA out of decisions regarding giving terminally ill patients access to experimental drugs has been rejected by the US House of Representatives. Although the FDA currently approves the majority of requests to try unapproved therapies in these circumstances, the bill sought to place the decision wholly in the hands of the patient’s healthcare provider and the treatment’s manufacturer.
The ‘right to try’ unapproved treatments, which often includes experimental cell-based therapies, has caused immense controversy with strong opinions on both sides of the debate. Proponents highlight stories of patients that were or could have been saved with expanded access. Opponents cite patients that have been irrevocably harmed through administration of unapproved treatments. With a similar bill already passed in the Senate, and 38 states, including Texas, having already signed bills enshrining the ‘right to try’, its clear the fight is not over.
Micronized amniotic tissue granted RMAT for the treatment of osteoarthritis
MiMedx Group, Inc. (GA, USA) has received a Regenerative Medicine Advanced Therapy (RMAT) designation for AmnioFix® Injectable for the indication of osteoarthritis. AmnioFix is an allogeneic micronized dehydrated human amnion/chorion membrane that is injected into the knee to reduce scar tissue formation, modulate inflammation and enhances healing.
“The FDA’s RMAT designation for AmnioFix Injectable is an important milestone for MiMedx and highlights the importance of introducing promising therapies in areas of considerable unmet medical need,” commented Parker H. “Pete” Petit, Chairman and Chief Executive Officer (MiMedx).
“AmnioFix Injectable has been shown to have the potential to reduce pain and improve function in patients with OA of the knee. Given the lack of treatment options for patients with this condition, and the high risks associated with using opioids to manage pain, AmnioFix Injectable has the opportunity to address a significant unmet clinical need by providing physicians and their patients with a new front-line therapy,” added Bill Taylor, President and COO (MiMedx).
Phase I/II Duchenne muscular dystrophy trial placed on clinical hold
Solid Biosciences Inc. (MA, USA) has confirmed that a phase I/II trial for its gene-transfer therapy for Duchenne muscular dystrophy, SGT-001, has been placed on clinical hold after a patient reported an unexpected serious adverse event. Following hospitalization of the patient after tests demonstrated a decrease in platelet count, a reduction in red blood cell count and evidence of complement activation, enrollment and dosing in the trial were halted. The patient responded well and is currently asymptomatic.
In a statement to the Duchenne community, Solid Biosciences explained: “Since our inception, we have been focused on working to bring meaningful treatments to patients with Duchenne muscular dystrophy….We greatly appreciate the trust placed in us by patients and families and will honor that through our dedication and work to end this disease. We appreciate your patience while we work to resolve this situation.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.