Current Pipeline

NGENLA™, a long-acting human growth hormone (hGH) molecule, is a once-weekly injectable, created using recombinant technology, for the treatment of children and adolescents with a growth hormone deficiency (GHD). The molecule consists of the natural peptide sequence of native growth hormone and the 28 amino acids of the CTP. This molecule, as compared to current GH replacement therapies, is intended to reduce the injection frequency from a daily to once a week in children and adolescents with GHD.

NGENLA™ Received Marketing Authorization for the treatment of Pediatric Growth Hormone Deficiency in the U.S., the European Union, UK, Canada, Australia, South Korea, Japan, Taiwan, India and Brazil.

Factor VIIa-CTP is a novel, long-acting recombinant Factor VIIa intended to be used to treat and to prevent bleeding episodes in hemophilia A or B patients. This molecule utilizes CTP technology to extend the circulatory half-life and significantly improves the treatment of hemophilia patients by reducing the frequency of rFVIIa infusions and by enabling prophylactic treatment.

Factor VIIa-CTP has been granted orphan drug designation in the U.S. and Europe.

Human growth hormone (hGH) antagonists are used to treat acromegaly, a disorder caused by excess human growth hormone. OPKO Biologics is developing a long acting hGH antagonist utilizing a stabilizer-based technology to extend its serum half-life and to reduce the frequency of injections, with the goal of reaching once weekly administration.

Parathyroid hormone (PTH) is used to treat hypoparathyroidism (HPT), a rare endocrine disorder caused by deficient or absent PTH hormone. PTH plays a critical role in controlling calcium, phosphate and vitamin D levels in the blood and bone. OPKO Biologics is developing a long acting PTH utilizing a stabilizer-based technology to extend its serum half-life and to reduce the frequency of injections, with the goal of reaching once weekly administration.

Severe primary insulin-like growth factor 1 (IGF-1) deficiency (SPIGFD) results in short stature and increased insulin sensitivity in afflicted patients. We are developing OPK-8801002, utilizing a stabilizer-based technology, as an extended release IGF-1 repletion therapy for the treatment of SPIGFD.

SPIGFD occurs in roughly 20% of children with isolated short stature.