Drug Review Profiles

Stemline Therapeutics’ Elzonris was approved for a rare cancer in Europe in January, following a long and arduous two-year journey through the EU regulatory process that included an initial rejection and a subsequent re-examination by the EMA. Its fortunes were very different in the US, where the review took just six months from filing to approval.

US FDA has not deviated from its bedrock trust in the value of ‘statistically reliable’ endpoints amidst the COVID-19 pandemic, the Pink Sheet’s Drug Review Profile of Gilead’s Veklury shows.

Just five and half years elapsed between Gilead’s first remdesivir IND for Ebola virus and Veklury’s US FDA approval for COVID-19.

Not everyone reviewing Daiichi Sankyo’s EU marketing application for Turalio agreed that the potential tenosynovial giant cell tumor treatment shouldn't be approved.

Agency approved Regeneron’s monoclonal antibody cocktail Inmazeb and Ridgeback’s mAB Ebanga on data from a multi-arm, adaptive trial in which several investigational agents were independently compared to a control group. Efficacy for both drugs was demonstrated on a mortality endpoint, with lingering uncertainties to be addressed through postmarketing studies.

Sponsor preferred a tropical disease voucher because it could be redeemed for a supplemental application, but the FDA awarded a voucher under the medical countermeasures program, which prohibits redemption for supplements; agency says it takes a case-by-case approach to deciding whether tropical disease and rare pediatric disease vouchers can be redeemed for supplements.

The individual contribution of the three mABs in Regeneron’s Ebola treatment could not be clinically tested due to the highly lethal nature of the disease and the potential for development of resistance; instead, the FDA accepted nonclinical data demonstrating the mechanism of action for each component.

Chronicle of the development and review of Regeneron’s REGN-EB3, a triple mAB combination treatment for Ebola infection.

Chronicle of the development and review of Ridgeback Biotherapeutics’ ansuvimab-zykl for treatment of Ebola infection.

The European public assessment report for Novartis's advanced breast cancer therapy, Piqray (alpelisib), includes some interesting insights into what EU regulators are looking for in drug company clinical trials. 

The European public assessment report for Kaftrio shows that EU regulators had concerns about whether Vertex’s triple drug combination for cystic fibrosis represented a new treatment paradigm.

Product review report reveals multiple delays and missteps on route to market, holding valuable lessons for other gene therapy developers but also evidencing an ultimately welcoming environment.