YONGIN, South Korea and AUSTIN, Texas, Aug. 30 2022 /PRNewswire/ — GC Biopharma (006280.KS) and Speragen today announced that a joint Externally Led – Patient-Focused Drug Development (EL-PFDD) meeting hosted by SSADH Association and attended by the U.S. FDA and other stakeholders was held to address the patient experience with SSADHD (Succinic Semialdehyde Dehydrogenase Deficiency), a disorder caused by mutations in the ALDH5A1 gene which is inherited in an autosomal recessive manner.
The purpose of this meeting is for patients affected by SSADHD, and their families, to provide clear perspectives to researchers, academics, industry partners, and the FDA on living with their condition and what defines a meaningful treatment. In the meeting, a total of 113 participations including patients, families, KOLs, members of FDA, GC Biopharma and Speragen, were gathered from all over the world.
The participants were communicating to the FDA about the impacts of SSADHD on individuals’ daily lives, what types of treatment benefits impact patients’ lives, and perspectives on how well available therapies are working.
Based on the discussion, the SSADH Association will submit the Voice of the Patient report to the FDA by the end of this year. GC Biopharma and Speragen also plan to develop a clinical outcome assessment for developing ERT (Enzyme Replacement Therapy) for SSADHD to meet the patient’s unmet needs.
"The meeting was a major step towards our ultimate goal of providing new treatment options to the patients and their families," said EC Huh, Ph. D., President of GC Biopharma. "We will continue our work to provide rare disease treatments and expand our R&D pipelines."
"We are so very proud of the patients and families for sharing their experiences and impacts from living with SSADHD. It took great vulnerability and courage to be so open about their struggles" said Alice McConnell, CEO of Speragen.
Succinic semialdehyde dehydrogenase deficiency (SSADHD) is a disorder that can cause a variety of neurological and neuromuscular problems. The signs and symptoms can be extremely variable among affected individuals and may include mild to severe intellectual disability; developmental delay (especially involving speech); hypotonia; sleep disturbances; difficulty coordinating movements (ataxia); and/or seizures. Some affected individuals may also have decreased reflexes (hyporeflexia); nystagmus; hyperactivity; and/or behavioral problems. SSADH deficiency is caused by mutations in the ALDH5A1 gene and is inherited in an autosomal recessive manner. Management is generally symptomatic and typically focuses on treating seizures and neurobehavioral issues. Like so many rare diseases, there is no treatment for SSADHD worldwide.
About GC Biopharma
GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century.
Speragen was co-founded by a mother of two children who are affected with SSADHD. Speragen knows first-hand the challenges of navigating a life with a rare disease: from the daily challenges, the diagnostic odyssey, accepting the new reality post-diagnosis, finding a team of knowledgeable clinicians who can provide care, and finally to seeing the chasm between the current state of treatment and the possibility of a first-in-class drug becoming available. Speragen, comes from the Latin word "spera" meaning "hope". It was formed to help solve these issues more rapidly by bridging the gulf from hope to the realization of a therapy.
This press release may contain forward-looking statements, which express the current beliefs and expectations of GC Biopharma’s management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.
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