Sanofi ALTUVIIIO hemophilia A treatment: FDA accepts sBLA based on pediatric phase 3 data

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The FDA has accepted Sanofi’s supplemental Biologics License Application (sBLA) for ALTUVIIIO, a novel, high-sustained factor VIII replacement therapy for adults and children with hemophilia A. The FDA based the decision on positive final data from the pivotal Phase 3 XTEND-Kids trial in children under 12 with hemophilia A.

ALTUVIIIO (efanesoctocog alfa) is the first Factor VIII replacement therapy to offer bleed protection with once-weekly dosing. In February, Sanofi announced that FDA had approved the drug for routine prophylaxis and on-demand treatment to control bleeding episodes. Its use also extends to perioperative management (surgery) for adults and children with hemophilia A.

Sanofi developed in collaboration with Sobi. Barclays projects that the drug will top €2 billion in peak sales.

Supportive findings from XTEND-kids study

The current FDA approval is supporte…

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FDA grants Enhertu priority review for HER2-positive positive metastatic breast cancer

FDA has accepted the supplemental Biologics License Application (sBLA) for Enhertu (trastuzumab deruxtecan), a HER2-directed antibody and DNA topoisomerase I inhibitor conjugate from AstraZeneca (LON:AZN) and Daiichi Sankyo (OTCMKTS:DSNKY).

The sBLA pertains to treating adults with unresectable or metastatic HER2-positive breast cancer previously treated with an anti-HER2-based regimen.

FDA also granted priority review to the application.

In 2019, Enhertu first won FDA approval for HER2-positive unresectable or metastatic breast cancer after two or more prior anti-HER2-based regimens.

Two years later, the agency extended approval to patients with previously-treated HER2-positive advanced gastric cancer.

The recent application includes data from the DESTINY-Breast03 study,

That study found that Enhertu demonstrated a statistically significant improvement in progression-free survival compared with trastuzumab emtansine (T-DM1) (Kadcyla…

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Pfizer-BioNTech COVID-19 vaccine 100% effective in adolescents 12 to 15

Pfizer (NYSE:PFE) and BioNTech (NSDQ:BNTX) shared new data from their Phase 3 study of the Comirnaty vaccine, concluding that two 30-µg doses were 100% effective against COVID-19 between seven days and four months after the second dose.

The safety profile of the vaccine in the study was broadly consistent with other clinical safety data. In addition, clinical trial investigators reported no serious safety concerns in participants in the six months following receipt of the second dose.

Data from 2,228 individuals were included in the analysis.

In the U.S., the Comirnaty vaccine is available under emergency use authorization for adolescents 12 to 15. The FDA approved the vaccine for individuals at least 16 years of age in August. In late October, the companies received emergency use authorization for the vaccine in children 5 to 11.

Pfizer and BioNTech plan on including the recent data in a planned supplemental Biologics License Application (sBLA) t…

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