Medical Device Daily Washington Writer

Valera Pharmaceuticals (Cranbury, New Jersey) has filed for FDA approval of Supprelin-LA, a 12-month implant for a condition known as central precocious puberty (CPP), the early onset of puberty.

The agency already has named the product an orphan drug for CPP, characterized by the premature development of secondary sexual characteristicsin young children due to increased secretion of sex hormones. A regulatory decision is expected next year.

The subcutaneous implant, which is inserted into the upper arm, employs the company's Hydron technology. Made of a soft, flexible material, it measures about an inch long and a quarter-inch wide. Its underlying technology is based upon specialty blends of hydrogel polymers spun into small tubes, as is Vantas, which also is a histrelin-based product, though of a different formulation with a slower release rate. It received FDA approval in October 2004.

The product provides a controlled dose of histrelin, a “very potent” synthetic nonapeptide agonist of naturally occurring gonadotropin-releasing hormone (GnRH), said Stuart Levine, Valera's director of investor relations. Standard treatments for CPP involve the use of such GnRH agonists to suppress hormonal production to delay the onset of puberty.

“This is a treatment option that we would be offering to not only the patients,” Levine said, “but also their parents, who have to subject their kids right now to monthly intramuscular injections.”

The company estimates that the annual CPP market tops $75 million in the U.S. Assuming approval for Supprelin-LA, Valera's existing sales representatives would market the product in addition to their duties for the company's approved implant for advanced prostate cancer, Vantas. Levine told Medical Device Daily's sister publication, BioWorld Today, that urologists targeted for Vantas operate in many of the same areas as pediatric endocrinologists that would be targeted for Supprelin-LA.

The new drug application was based largely on Phase III results from an open-label study of 36 patients ranging in age from 4 to 11. Of them, 16 had received GnRH therapy prior to enrollment while the remainder was treatment na ve. Though specific data have not been released, preliminary results showed that treatment with Supprelin-LA led to rapid sex hormone suppression that was sustained for a year.

“We're very encouraged by the data,” Levine said, noting also the product's convenience. “We think the potency and efficacy look good.”

The study's primary endpoints were hormonal suppression below pubertal levels and continued suppression upon challenge with GnRH.

If approved, Supprelin-LA would compete with Lupron Depot-PED (leuprolide acetate) from TAP Pharmaceutical Products. That monthly injectable product, Levine said, is the primary standard of care in this space, which includes about 15,000 patients in the U.S.

A daily injection of Supprelin received FDA approval in the 1990s as an orphan drug. Valera acquired rights to the brand name earlier this year.

“I'm personally not aware of the FDA giving orphan drug status to the same chemical entity twice,” Levine said, “so we're feeling good about this particular product and the filing.”

Typically, CPP hormonal therapy could run three to five years or more, depending on a child's age upon diagnosis. If left untreated, it can cause short stature because of premature bone plate fusion, in addition to social and psychological problems. The condition is more common in girls, often manifesting in such secondary sexual characteristics as early menstruation or breast development.

Valera, which focuses on products to treat urology and endocrinology diseases and disorders, expects to soon begin marketing a recently acquired drug therapy for certain urinary bladder cancer patients called Valstar. In addition, its R&D efforts are centered on applying its core Hydron technology to other known compounds.

The company went public earlier this year, raising about $30 million at a per-share price of $9.