Krystal Biotech Announces Home Dosing in B-VEC Open Label Extension Study
Krystal Biotech, Inc. recently announced it plans to offer patients with dystrophic epidermolysis bullosa (DEB), who are enrolled in the GEM-3 open label extension study, the opportunity to be dosed in their homes by a health care professional (HCP). Krystal is offering this convenience to patients based on feedback from the US FDA following review of Krystal’s human factors (HF) validation study report submitted in January 2022.
“We thank the Agency for their consideration and believe that dosing patients in the comfort of their homes relieves them from an undue burden of travel and improves compliance with the therapy,” said Suma Krishnan, Founder and President of Research & Development at Krystal.
Per the FDA’s guidance, Krystal plans to address the convenience of dosing a patient at home of our intend-to-market B-VEC commercial product in its BLA submission that is anticipated in 2Q, 2022.
The GEM-3 B-VEC OLE study is a 78-week (approximately a year and a half) open-label extension study of B-VEC for participants diagnosed with DEB who are aged 6 months and older that began in May 2021. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study will enroll subjects who participated in GEM-3 Phase 3 study, as well as new participants who were unable to participate in the Phase 3 study and who meet all enrollment criteria.
B-VEC is an investigational non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. B-VEC was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism.
The FDA and EMA have each granted B-VEC orphan drug designation for the treatment of DEB, and the FDA has granted B-VEC fast track designation and rare pediatric designation for the treatment of DEB. In addition, in 2019, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) to B-VEC for the treatment of DEB and the EMA granted PRIority MEdicines (PRIME), eligibility for B-VEC to treat DEB.
Krystal Biotech, Inc. is a pivotal-stage gene therapy company leveraging its proprietary, redosable gene therapy platform and in-house manufacturing capabilities to potentially bring life-changing treatment options to patients with serious diseases, including rare diseases in skin, lung, and other areas. For more information, visit http://www.krystalbio.com.
Total Page Views: 821
