Products targeting rare diseases are getting lots of publicity following Shire Pharmaceuticals’ (SHRP) announced acquisition of ViroPharma (VRPH), a rare-disease biopharmaceutical company, for $4.2 billion.
At the American Society of Nephrology (ASN) annual meeting, Alexion Pharmaceuticals (ALXN) presented data from four clinical trials showing positive benefits for Soliris, its treatment for atypical hemolytic uremic syndrome (aHUS). For PropThink’s latest assessment of ALXN, see Ivan Deryugin’s summer update.
Soliris is the first and only approved safe and effective treatment for pediatric and adult patients with aHUS, a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death. The condition causes the body to destroy red blood cells (hemolysis) and create clots in small blood vessels (TMA) that can destroy kidney function. The condition can arise at any time and is estimated to occur in 1 of 500,000 people, or approximately 600-700 cases per year in the U.S. alone. Sixty-five percent of all patients with aHUS die, require kidney dialysis, or have permanent kidney damage within the first year after diagnosis. The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent problems arising from aHUS, resulting in a very high level of transplant failure.
In the most important trial, researchers shared positive new data from the largest prospective trial of Soliris. The trial enrolled 41 adult patients with aHUS representing a broad patient population. The study looked at patients after 26 weeks of treatment. The primary endpoint of the study was the proportion of patients with complete TMA response, as measured by platelet count normalization, LDH normalization and preservation of renal function (<25% increase in serum creatinine from baseline), at 26 weeks. The study met its primary endpoint, with 30 of 41 patients (73%) achieving a complete TMA response at 26 weeks. Forty of 41 patients (98%) achieved platelet count normalization by week 26. Soliris significantly improved renal function with a mean increase in eGFR. Most importantly, of the 24 patients who were on dialysis at baseline, 20 patients (83%) discontinued dialysis by week 26.