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FDA Approves DARZALEX FASPRO® (daratumumab & hyaluronidase-fihj) + Carfilzomib & Dexamethasone for Multiple Myeloma Patients After 1st or Subsequent Relapse
“Data from the PLEIADES trial continue to support additional treatment combinations that can influence the course of this disease as early as after the first relapse by providing durable responses that may help to delay progression,” said Ajai Chari, M.D.*, Professor of Medicine, Director of Clinical Research in the Multiple Myeloma Program, and Associate Director of Clinical Research in the Mount Sinai Cancer Clinical Trials Office. “The approval of subcutaneous daratumumab in combination with Kd will help clinicians address unmet patient needs by reducing the administration time from hours to just minutes, and reducing the frequency of infusion-related reactions, as compared to the intravenous daratumumab formulation in combination with Kd.” 

U.S. Withdrawal of FARYDAK ® (panobinostat) NDA Announced

• Based on discussions with the U.S. FDA, Secura Bio has submitted to FDA for the withdrawal of the approval of NDA 205353 for FARYDAK® (panobinostat) oral capsules. FARYDAK received accelerated approval in February 2015 for use in combination with bortezomib and dexamethasone to treat patients with multiple myeloma who have received at least two prior regimens, including bortezomib and an immunomodulatory agent. Secura Bio acquired FARYDAK in March 2019.
• The accelerated approval was based on PFS and consistent with FDA regulations, required further adequate and well-controlled clinical studies to verify and describe the product’s clinical benefit. In its withdrawal submission, Secura Bio noted that, as previously discussed with FDA, it was not feasible for the company to complete the required post-approval clinical studies as designed as part of the accelerated approval process. Because those studies were required to verify and describe the clinical benefit of the drug product, the clinical benefit of FARYDAK has not been confirmed under the specific constraints of the accelerated approval process.
• Because of the withdrawal submission, FARYDAK will no longer be discussed at the December 2, 2021 meeting of the Oncologic Drugs Advisory Committee. As provided for by FDA regulations, Secura Bio anticipates FDA publishing a Federal Register notice announcing withdrawal of the approval.

Development of rebastinib to be discontinued
“The decision to realign our resources and restructure our organization was difficult, but one which will allow us to focus on the critical programs that will drive our future growth. I would like to personally express my appreciation to our colleagues who are impacted by this decision. We are immensely grateful for their dedication and their contributions to advancing our mission,” said Steve Hoerter, President and Chief Executive Officer of Deciphera. “We remain excited by the strength of our pipeline and the opportunity for QINLOCK to continue to benefit patients with advanced GIST. We have a clear and positive path forward with a committed team that is fully invested in the future of Deciphera.”

Krystal Biotech announces positive results from gene therapy trial for rare skin disease
Krystal Biotech announced positive topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as VYJUVEK^TM, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB). VYJUVEK^TM is an investigational non-invasive, topical gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to dystrophic EB wounds. Dystrophic EB is a rare and severe monogenic disease caused by a mutation in the COL7A1 gene. The lack of a functional COL7A1 protein leads to absence of anchoring fibrils which causes extremely fragile skin that blisters and tears from minor friction or trauma.