FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Cystinosis Treatment Granted Priority Review Status

Sigma-Tau Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted the filing of the company's new drug application (NDA) for its cysteamine hydrochloride ophthalmic solution, an investigational therapy for the treatment of corneal cystine crystals in patients with cystinosis. The FDA also granted the NDA Priority Review status, a designation given to drugs that may provide major advances or a treatment where no adequate therapy exists.

Cystinosis is a rare disease affecting as few as 300 people in the U.S. The company has been granted orphan drug designation for this product and previously received a grant from the FDA's Orphan Drug Products Division to help fund the treatment research.  If approved, this medicine will be the first FDA approved treatment for corneal cystine crystal accumulation, which can lead to changes in visual acuity and photophobia (i.e., sensitivity to light).  

"Acceptance of the NDA marks an important milestone for both Sigma-Tau and the cystinosis community," said Gregg Lapointe, Chief Executive Officer of Sigma-Tau Pharmaceuticals.  "If approved, this treatment has the potential to offer important benefits for patients—many of whom are children—whose care has been limited due to a lack of therapeutic options and access."

The NDA includes data from clinical trials conducted at the National Institutes of Health (NIH) National Eye Institute.  Results of the studies indicate that administration of cysteamine hydrochloride eye drops may be effective in the prevention and treatment of corneal cystine crystals.

"Cystinosis is a devastating disease that too often robs children of normal vision," said Christy Greeley, President and Executive Director of the Cystinosis Research Network, Inc.  "We are excited about the prospect of a new, accessible treatment option for our kids."

SOURCE Sigma-Tau Pharmaceuticals, Inc.