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Roche Gets Drug Approvals for First Treatment for a Rare Blood Disorder and NSCLC


Roche had 2 drugs approved by the FDA—one that is the first approved treatment for a rare blood disease and the other is approved as a first-line treatment for anaplastic lymphoma kinase-positive metastatic non–small cell lung cancer.

Roche had 2 drugs approved by the FDA—one for a rare blood disease and the other for first-line treatment for lung cancer.

Zelboraf (vemurafenib) is the first FDA-approved drug for Erdheim-Chester disease (ECD), a rare blood disorder. The drug is already approved for the treatment of people with unresectable or metastatic melanoma with the BRAF V600E mutation. The FDA has now approved it specifically for patients with ECD who have the BRAF V600 mutation. ECD is characterized by the abnormal multiplication of histiocytes. These white blood cells can invade normal tissues and organs.

The drug was approved based on date from the phase 2 VE-BASKET study, which used the innovative clinical trial design matched a disease’s underlying genetic profile to the mechanism of action of the medicine. For the 22 people with ECD, the trial showed a best overall response rate of 54.5%.

“This FDA decision means people living with Erdheim-Chester disease will now, for the first time, have an FDA-approved treatment option,” Sandra Horning, MD, Roche’s chief medical officer and Head of Global Product Development, said in a statement. “We are committed to finding new ways to bring medicines to patients with high unmet need, and we are pleased that this innovative clinical trial helped identify Zelboraf for treatment of this rare disease.”

The second drug approved was Alecensa (alectinib) as a first-line treatment for people with anaplastic lymphoma kinase (ALK)-positive metastatic non—small cell lung cancer (NSCLC). The FDA approved the drug based on results from the phase 3 ALEX study, which showed the drug reduced the risk of disease worsening or death by 47% compared with crizotinib. Median progression-free survival was 25.7 months for people on alectinib compared with 10.4 months for those on crizotinib.

Alectinib has been recommended in the National Comprehensive Cancer Network guidelines as a treatment option for first-line ALK-positive metastatic NSCLC.

“Our goal is to develop medicines that have the potential to significantly improve upon the standard of care,” Horning said in a separate statement. “In our pivotal study, Alecensa significantly extended the time that people lived without their disease worsening compared to crizotinib and also showed a marked reduction in the risk of their cancer spreading to the brain.”

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