FDA Approves Regeneron & EUSA Drugs

Regeneron Pharmaceuticals and EUSA Pharma have announced that US regulators have approved their blindness drug and leukaemia therapy, respectively.

The US FDA (Food and Drug Administration) has given the okay to Regeneron’s Eylea, also known as VEGF Trap-Eye, for the treatment of patients with wet age-related Macular Degeneration at a recommended dose of 2 milligrams every four weeks for the first 12 weeks, followed by 2 mg every eight weeks.

Age-related macular degeneration (AMD) is a principal cause of acquired blindness.  Macular degeneration is diagnosed as either dry (non-exudative) or wet (exudative).  In wet AMD, new blood vessels grow below the retina and leak blood and fluid.  This leakage results in disruption and dysfunction of the retina creating distortion and/or blind spots in central vision.  Wet AMD is the primary cause of blindness for people over the age of 65 in the U.S. and Europe.

The approval, granted under priority review, is based on two Phase III trials which showed that Eylea was clinically equivalent to the standard of care of Roche’s Lucentis (ranibizumab injection).

The most common adverse reactions reported in patients receiving Eylea were conjunctival hemorrhage, eye pain, cataract, vitreous detachment, vitreous floaters, and increased intraocular pressure.  The adverse event profile was similar to that seen with ranibizumab.

Jeffrey Heier from Tufts School of Medicine, and Chair of the Steering Committee for the VIEW 1 trial, commented that the approval “offers a much needed new treatment option for patients with wet AMD”. He added that Eylea achieved “the efficacy we’ve come to expect from current anti-VEGF agents, but with less frequent injections and no monitoring requirements. This may reduce the need for costly and time-consuming monthly office visits for patients and their caregivers”.

Meanwhile, the FDA also gave approval to EUSA’s orphan drug, Erwinaze, for the treatment of acute lymphoblastic leukaemia in patients with hypersensitivity to existing E coli-derived versions of the treatment.

Acute lymphoblastic leukaemia is the most common form of child cancer, with approximately 2,900 patients under the age of 20 diagnosed in the USA each year. It is one of the most curable forms of cancer, with remission rates in treated children of over 95% and 75 – 85% surviving at least five years without recurrence.

“Treatment with asparaginase is a vital and life-saving therapy for thousands of patients, mostly children, with acute lymphoblastic leukemia each year. Unfortunately, a number of these patients develop hypersensitivity to asparaginases derived from E. coli, including pegaspargase, and are unable to complete the recommended course of treatment. The approval of ERWINAZE is an important advance because it is the only treatment option that can enable these patients to continue and complete their full course of therapy,” said Stephen Sallan MD, Chief of Staff, Dana-Farber Cancer Institute and Professor of Pediatrics, Harvard Medical School.  EUSA noted that an estimated 15%-20% of acute lymphoblastic leukaemia patients develop hypersensitivity to E coli-derived asparaginase.

Chief executive officer of EUSA Pharma, Bryan Morton, commented that “launching Erwinaze to sit alongside our existing portfolio of specialty products is also a major strategic milestone for the company, as this is the first treatment Eusa has developed internally”.

The Erwinaze approval is based on the results of clinical studies in 630 acute lymphoblastic leukaemia patients. In the pivotal efficacy study, conducted in 58 subjects, 100% of evaluable patients achieved the asparaginase activity primary endpoint.



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