Despite the U.S. FDA’s approval of Sage Therapeutics Inc.’s priority NDA for postpartum depression, the accompanying complete response letter (CRL) for major depressive disorder has undercut the company’s plans. Withholding the approval for MDD slices away a huge amount of the potential market for Sage and its collaborator, Biogen Inc.
Regenerative medicine company Mesoblast Ltd.’s stock sank nearly 57% on the news that it received a second U.S. FDA complete response letter (CRL) following the resubmission of its BLA for allogeneic stem cell treatment remestemcel-L in children with steroid-refractory acute graft-vs.-host disease. In the CRL, issued a few days after the Aug. 2 PDUFA date, the agency said it requires more data to support approval.
The U.S. FDA issued a complete response letter regarding the BLA for Immunitybio Inc.’s bladder cancer treatment, N-803 (Anktiva), halting the drug’s development and slicing the stock value in half. The problems stem from the FDA’s pre-license inspection of Immunitybio’s third-party contract manufacturing organizations, the company said. It said the FDA also had recommendations for specific chemistry, manufacturing and controls issues and assays that needed to be resolved before the BLA can be approved.
It’s not often that getting an NDA rejected by the U.S. FDA is cause for a company’s stock to rise. But in the case of Ascendis Pharma A/S, the agency’s anticipated complete response letter (CRL) for Transcon PTH (palopegteriparatide) offered some much-needed clarity and a potentially optimistic time frame for an NDA resubmission seeking approval of the once-daily hormone replacement therapy for hypoparathyroidism, news that sent the company’s shares (NASDAQ:ASND) up 24%, or $16.78, to close May 1 at $86.74.
After receiving a U.S. FDA complete response letter nearly two years ago, Cyclopharm Ltd. said the agency has accepted its response and reset the clock for the NDA review of its Technegas combination product for pulmonary embolisms, with a new PDUFA date set for Sept. 29.
After receiving a U.S. FDA complete response letter nearly two years ago, Cyclopharm Ltd. said the agency has accepted its response and reset the clock for the NDA review of its Technegas combination product for pulmonary embolisms, with a new PDUFA date set for Sept. 29.
After receiving a U.S. FDA complete response letter nearly two years ago, Cyclopharm Ltd. said the agency has accepted its response and reset the clock for the NDA review of its Technegas combination product for pulmonary embolisms, with a new PDUFA date set for Sept. 29.
The U.S. FDA has accepted Mesoblast Ltd.’s BLA resubmission for its allogeneic stem cell treatment remestemcel-L in children with steroid-refractory acute graft-vs.-host disease (SR-aGVHD). Mesoblast received a complete response letter from the FDA in October 2020 for remestemcel-L even though approval was highly anticipated after the FDA’s Oncologic Drugs Advisory Committee voted 9-1 that the stem cell therapy showed evidence of efficacy as a treatment for SR-aGVHD in children.
The U.S. FDA has accepted Mesoblast Ltd.’s BLA resubmission for its allogeneic stem cell treatment remestemcel-L in children with steroid-refractory acute graft-vs.-host disease (SR-aGVHD). Mesoblast received a complete response letter from the FDA in October 2020 for remestemcel-L even though approval was highly anticipated after the FDA’s Oncologic Drugs Advisory Committee voted 9-1 that the stem cell therapy showed evidence of efficacy as a treatment for SR-aGVHD in children.
The U.S. FDA issued Cytokinetics Inc. a complete response letter (CRL) for omecamtiv mecarbil, branding the clinical evidence behind it as not “persuasive” enough to establish its effectiveness for reducing the risk of heart failure events and cardiovascular death. The company is developing the drug as an add-on therapy for patients with worsening heart failure who remain at high risk for heart failure events and hospitalization.
