Rare Daily Staff

The U.S. Food and Drug Administration granted accelerated approval to Bayer Healthcare’s Aliqupa, a treatment for adults with relapsed follicular lymphoma who have received at least two prior systemic therapies.

Follicular lymphoma is a slow-growing type of non-Hodgkin lymphoma, a cancer of the lymph system. The lymph system is part of the body’s immune system and is made up of lymph tissue, lymph nodes, the spleen, thymus, tonsils and bone marrow. A little more than 72,000 people a year in the United States will be diagnosed with some form of non-Hodgkin lymphoma this year with more than 20,000 expected to die from the disease in 2017.

Aliqopa is a kinase inhibitor that works by blocking several enzymes that promote cell growth. Under accelerated approval, the FDA relies on clinical data that is believed to be predictive of clinical benefit for a drug that addresses unmet medical needs for serious conditions, but requires confirmation of clinical benefit through additional studies, which Bayer is conducting.

“When treating patients with relapsed follicular lymphoma, two factors for physicians involve lessening tumor burden and reducing adverse events patients may experience from treatments. Therapies need to be both effective and tolerable,” said Anas Younes, Medical Oncologist and Chief of Lymphoma Service at Memorial Sloan Kettering Cancer Center. “The PI3K pathway is activated in many tumor types, including lymphoma, and targeting PI3K therapeutically is a very important strategy in this area of disease.”

The agency granted Aliqopa approval based on data from a single-arm trial that included 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. The trial measured how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). In the trial, 59 percent of patients had a complete or partial response for a median 12.2 months.

Common side effects of Aliqopa include high blood sugar levels, diarrhea, decreased general strength and energy, high blood pressure, low levels of certain white blood cells, nausea, lower respiratory tract infections, and low levels of blood platelets.

Serious side effects include infections, high blood sugar levels, high blood pressure, inflammation of the lung tissue, low levels of certain white blood cells, and severe skin reactions. Women who are pregnant or breastfeeding should not take Aliqopa because it may cause harm to a developing fetus or newborn baby.

The agency had previously granted Aliqopa Priority Review designation, which calls for the FDA to act on an application within six months, and Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

September 15, 2017

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