FDA approves a Scholar Rock BLA for Isembyld (apitegromab-mstn) injection to treat spinal muscular atrophy.
FDA seeks input on whether traditional statistical standards should be modified for rare disease clinical investigations, including when the agency might accept different significance thresholds, external controls and other approaches intended to address the challenges of studying very small patient populations.
FDA approves a Scholar Rock BLA for Isembyld (apitegromab-mstn) injection to treat spinal muscular atrophy.
FDA seeks input on whether traditional statistical standards should be modified for rare disease clinical investigations, including when the agency might accept different significance thresholds, external controls and other approaches intended to address the challenges of studying very small patient populations.
FDA’s approval of Outlook Therapeutics’ Lytenava shows that the agency’s willingness to accept one adequate and well-controlled trial plus confirmatory evidence can extend beyond rare diseases
AstraZeneca and Daiichi Sankyo says that Enhertu cut the risk of disease progression or death by 37% compared with pembrolizumab (Merck’s Keytruda) plus chemotherapy as a first-line treatment for patients with advanced HER2-mutant non-small cell lung cancer.
