December 16, 2014
By Jessica Wilson, BioSpace.com Breaking News Staff
Swiss pharmaceutical giant Novartis AG today announced that the U.S. Food and Drug Administration (FDA) has followed in the footsteps of the European Commission by approving the company’s rare endocrine drug Signifor long-acting release (LAR) for the treatment of acromegaly. The drug is approved for patients who have not responded adequately to the current standard of care for the disease.
An extremely rare endocrine disorder, acromegaly is caused by a non-cancerous growth in the pituitary gland that secretes an excess of growth hormone (GH). Effects of prolonged exposure to GH include the enlargement of hands, feet and facial features and a two- to three-fold increase in mortality.
“With the approval of Signifor LAR, physicians now have a new acromegaly therapy that provides an enhanced mechanism to address elevated hormone levels. This is a significant achievement and much welcomed news for patients with acromegaly,” said Monica Gadelha, a professor at Federal University of Rio de Janeiro and pivotal trial study author, in a statement.
Signifor LAR is a next-generation somatostatin analog (SSA) that binds to somatostatin receptors (SSTRs) and has the potential to stimulate both SSTR2 and SSTR5 subtype receptors to inhibit the secretion of GH. The approval of Signifor LAR is based upon Phase III trials that showed the drug was more effective in achieving biochemical control of acromegaly than the currently used SSAs octreotide LAR and lanreotide Autogel.
“The FDA approval of Signifor LAR for acromegaly marks an important day for physicians and patients living with difficult-to-treat pituitary conditions and underscores our continued commitment to helping patients manage rare diseases,” said President of Novartis Oncology Bruno Strigini in a statement.
Acromegaly affects approximately 60 people per one million, with three to four new cases diagnosed annually per one million people, according to Novartis. The company also stated that recent studies indicate the prevalence of acromegaly may be as high as 115 to 295 cases per million.
An important factor in treatment is that the disease is often not diagnosed for years because acromegaly is uncommon and the physical changes occur gradually, according to the Mayo Clinic. If not treated early, acromegaly becomes life threatening. Besides the symptoms of enlarged hands, feet and facial features, a patient can develop heart disease, hypertension, diabetes, arthritis and colon cancer. Heart disease is actually responsible for approximately 60 percent of deaths among people with acromegaly, according to Novartis.
Because acromegaly affects fewer than 200,000 people in the U.S., the FDA granted Signifor LAR an orphan drug designation. The orphan drug designation provides tax credits and other incentives for a company to develop drugs for rare diseases, for which the company would likely not recover the costs of the development of the drug.
