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Genentech scored two approvals from the FDA this week. Twice on Thursday, Genentech , scored two approvals from the U.S. Food and Drug Administration – a new medicine for hemophilia and an expansion for its cancer drug Gazyva . The regulatory agency approved Genentech’s hemophilia A drug Hemlibra and also approved Gazyva for patients with previously untreated advanced follicular lymphoma. The label expansion is for patients with stage II bulky, III or IV disease, the FDA said in announcing the label expansion. The new FDA approval has Gazyva being used in combination with chemotherapy followed by Gazyva alone for the common blood cancer. Follicular lymphoma, the most common slow-growing (indolent) form of non-Hodgkin’s lymphoma (NHL), is incurable and becomes harder to treat each time it returns. The FDA gave its nod to the label expansion based on the Phase III Gallium study. In that trial, Gazyva showed superior progression-free survival (PFS) for patients who received this Gazyva-based regimen compared with those who received a Rituxan (rituximab)-based regimen as an initial therapy. Gazyva is an engineered monoclonal antibody designed to attach to CD20, a protein found on certain types of B-cells. It is thought to work by attacking targeted cells both directly and together with the body’s immune system. In August, the FDA granted priority review for Gazyva following Genentech’s supplemental Biologics License Application. Genentech Chief Medical Officer Sandra Horning said the approval of Gazyva is important for those thousands of patients diagnosed with follicular lymphoma “who hope to delay disease progression for as long as possible.” Horning added that Gazyva is a treatment option that is shown to improve upon the company’s own drug Rituxan, which has become a standard –of-care treatment for the disease. With this approval, Gazyva is available in the United States for three different indications across two common types of blood cancer. The FDA first approved Gazyva in 2013 for people with previously untreated chronic lymphocytic leukemia (CLL). Last year, the FDA also approved Gazyva in combination with bendamustine followed by Gazyva alone as a treatment for people with follicular lymphoma who did not respond to a Rituxan-containing regimen, or whose follicular lymphoma returned after such treatment. Also yesterday, Genentech saw the approval of its hemophilia A drug Hemlibra which will be used to prevent or reduce the frequency of bleeding episodes in certain patients. Hemlibra will be released with a boxed warning for possible blood clots in patients. The annual hemophilia market is about $11 billion and Genentech hopes its indications of Hemlibra will secure a chunk of that market. With approval of Hemlibra, Genentech will rival drugs FEIBA and NovoSeven, developed by Shire Pharmaceuticals and Novo Nordisk respectively, for a share of the market.

The drug will be used to prevent or reduce the frequency of bleeding episodes in certain patients. The U.S. Food and Drug Administration approved Genentech ’s hemophilia drug Hemlibra (emicizumab-kxwh), which will be used to prevent or reduce the frequency of bleeding episodes in certain patients. Genentech , a division of Swiss drug giant Roche , said Hemlibra will soon be commercially available , but did not provide a timeline. The drug was reviewed by the FDA under a priority review. Hemlibra will be released with a boxed warning that concerns possible blood clots in patients, the FDA said. During trials, the clots were seen in patients who were “also given a rescue treatment (activated prothrombin complex concentrate) to treat bleeds for 24 hours or more while taking Hemlibra.” In October 2016, it was reported that four patients taking part in the trial suffered serious adverse events. Roche disclosed two of the patients developed thromboembolic events and two patients developed thrombotic microangiopathy. Genentech said nearly one-third of hemophilia A patients can develop antibodies known as factor VIII inhibitors. During clinical trials, Hemlibra was shown to substantially reduce bleeds in adults and children who have hemophilia A with the inhibitors. In the Phase III HAVEN 1 study, hemophilia A patients 12 years of age or older who received Hemlibra prophylaxis had a statistically significant reduction in treated bleeds of 87 percent. Similar results were seen in the HAVEN 2 study in children younger than age 12. In that study, 87 percent (of children who received Hemlibra prophylaxis) experienced zero treated bleeds. Sandra Horning , Genentech’s chief medical officer, said the approval of Hemlibra is an important advancement for this type of hemophilia patients who have struggled with being able to manage their bleeding issues. She said Hemlibra is the first type of medication to address this in nearly 20 years. In its announcement, Genentech released statements from some Hemlibra patients. Amber Hill, the mother of a 7-year-old hemophilia A patient with inhibitors, said before Hemlibra, her son needed intravenous infusions that could take up to two hours at least three times a week. Hill said the family’s life revolved around their son’s treatment. Now with Hemlibra, he can administer the medication himself, she said. With the newly approved drug he has fewer bleeds and has a greater quality of life, Hill said. “Reducing the frequency or preventing bleeding episodes is an important part of disease management for patients with hemophilia. Today’s approval provides a new preventative treatment that has been shown to significantly reduce the number of bleeding episodes in patients with hemophilia A with Factor VIII inhibitors,” Richard Pazdur , acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation, said in a statement. “In addition, patients treated with Hemlibra reported an improvement in their physical functioning.” Hemophilia A is a genetic blood-clotting disorder that can lead to uncontrolled bleeding. There are about 20,000 people in the United States diagnosed with hemophilia and 320,000 worldwide. Hemophilia A is the most common form. People with hemophilia A either lack or do not have enough of a clotting protein called factor VIII. The bleeding, which often occurs in joints, can cause significant pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage. The annual hemophilia market is about $11 billion and Genentech hopes its indications of Hemlibra will secure a chunk of that market. With approval of Hemlibra, Genentech will rival drugs FEIBA and NovoSeven, developed by Shire Pharmaceuticals and Novo Nordisk respectively, for a share of the market.