Solid has provided a letter to the community with the latest news on their halted IGNITE DMD program due to a serious adverse event experienced by the first patient dosed with SGT-001. While the patient continues to do well, Solid has been able to successfully resolve the previously-announced partial clinical hold on the planned high dose of SGT-001 in IGNITE DMD, which was due to manufacturing-related questions from the FDA. While encouraging, IGNITE DMD remains on hold until they address the full clinical hold as mentioned above.
Read the Letter to the Duchenne Community from Solid:
Dear Duchenne Community Members,
As valued partners in the fight against Duchenne muscular dystrophy (DMD), we wanted to share with you an update on our ongoing interactions with the U.S. Food and Drug Administration (FDA) on IGNITE DMD, our Phase I/II clinical trial to assess the safety and efficacy of SGT-001 in patients with DMD. We also shared this update in a press release that can be found here.
As you know, we recently announced that the FDA put IGNITE DMD on full clinical hold due to a serious adverse event (SAE) experienced by the first patient dosed with SGT-001. We reported the event to the FDA and have recently received the formal clinical hold letter, which outlined the agency’s additional information requests. We are working tirelessly to develop a comprehensive plan to address these questions and are committed to maintaining dialogue with the FDA with the hope of resuming IGNITE DMD as soon as possible. Most importantly, we’re pleased to say the treating physician has reported the patient is doing well.
As part of our update, we are also happy to share that we have been able to successfully resolve the previously-announced partial clinical hold on the planned high dose of SGT-001 in IGNITE DMD, which was due to manufacturing-related questions from the FDA. While encouraging, IGNITE DMD remains on hold until we address the full clinical hold as mentioned above.
Solid is deeply committed to advancing our mission to solve DMD. Our goal is and always has been to bring forward only the programs that we believe have life-changing potential and develop a portfolio of treatments to benefit all patients, regardless of their underlying genetic mutation, age or stage of disease. We knew this wasn’t going to be easy, but our mission and the possibility of helping those affected by this cruel disease guide every decision we make and project we take on.
We appreciate the Duchenne community’s continued support. We will provide additional updates in the future.
The Solid Team
Read the Press Release from Solid:
CAMBRIDGE, Mass., April 18, 2018 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB) today announced that the Company has received a letter from the U.S. Food and Drug Administration (FDA) relating to the previously-announced full clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational gene therapy, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). The Company plans to submit a response to this letter to the FDA in the coming weeks. The FDA also informed Solid that the Company has satisfactorily addressed manufacturing-related questions that led to the previously-announced partial clinical hold on the high dose of SGT-001 in IGNITE DMD.
“We believe SGT-001 has the potential to significantly benefit patients with DMD. We are working with the FDA to better understand the unexpected event that resulted in the clinical hold and put a plan in place to monitor and manage potential events in the future. I am happy to share that the treating physician has reported the patient is doing well,” said Ilan Ganot, Chief Executive Officer of Solid Biosciences. “We are also pleased that we were able to lift the earlier partial clinical hold on the high dose of SGT-001 through our existing manufacturing process, which is an important ongoing development effort for our company.”
The full clinical hold was in response to the Company’s report of a Serious Adverse Event (SAE) in the first patient dosed with SGT-001 in IGNITE DMD, where several days after administration the patient was hospitalized due to laboratory findings that included a decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation. The patient showed no signs or symptoms of coagulopathy and no relevant changes from baseline in liver function tests. Following medical treatment, all of his laboratory parameters have either improved or returned to normal.
In its full clinical hold letter, the FDA requested additional information that is required prior to resuming the clinical trial, including an assessment of the underlying etiology of the event, the patient’s clinical status and laboratory parameters, and any additional measures to address patient safety. Solid will work closely with the FDA to address these questions.
The earlier partial clinical hold on IGNITE DMD was related to manufacturing processes for the high dose of SGT-001. Solid submitted additional data to the FDA demonstrating that the current manufacturing process and product attributes for SGT-001 could support the high-dose group, enabling the use of a single lot for dose administration and limiting the number of vials of product required to treat each patient. The FDA notified Solid that its response has been accepted, and the partial clinical hold is now resolved.