Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Founded in 1996, the Company is publicly traded on the NASDAQ Global Market under the ticker symbol SBBP.
The primary focus of Strongbridge Biopharma is to build and advance a portfolio of therapeutically aligned rare disease franchises. Strongbridge’s rare endocrine franchise includes RECORLEV® (levoketoconazole), an adrenal steroidogenesis inhibitor currently being developed for the treatment of endogenous Cushing’s syndrome, and veldoreotide extended release, a preclinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both RECORLEV® and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. A new drug application (NDA) was submitted to the FDA for RECORLEV® in March 2021. The Company’s rare neuromuscular franchise includes KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. KEVEYIS® has orphan drug exclusivity in the United States.
The Company will continue to identify and evaluate the acquisition of products or product candidates that would be complementary to its existing rare endocrine and neuromuscular franchises or that would form the basis for new rare disease franchises.
Strongbridge Biopharma’s Product Portfolio
RECORLEV® (levoketoconazole) is an investigational adrenal steroidogenesis inhibitor in development for the treatment of patients with endogenous Cushing’s syndrome, a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. Levoketoconazole is the pure 2S,4R enantiomer derived from ketoconazole, a steroidogenesis inhibitor. RECORLEV® has demonstrated in two successful Phase 3 studies to significantly suppress serum cortisol and has the potential to be a next-generation adrenal steroidogenesis inhibitor.
The Phase 3 program for RECORLEV® includes SONICS and LOGICS: two multinational studies designed to evaluate the safety and efficacy of RECORLEV® when used to treat endogenous Cushing’s syndrome. The SONICS study met its primary and secondary endpoints, demonstrating a statistically significant normalization rate of urinary free cortisol at six months. The LOGICS study, which met its primary endpoint is a double-blind, placebo-controlled randomized-withdrawal study of RECORLEV® that is designed to supplement the long-term efficacy and safety information supplied by SONICS. The ongoing long-term open-label OPTICS study will gather further useful information related to the long-term use of RECORLEV®.
Levoketoconazole has received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing's syndrome. A new drug application (NDA) was submitted to the FDA for RECORLEV® in March 2021.
Veldoreotide extended release, a preclinical next-generation somatostatin analog, is under evaluation in nonclinical disease models amenable to somatostatin receptor modulation. Strongbridge has completed the screening of potential long-acting release (LAR) technologies for veldoreotide, and selected a proprietary formulation based upon PLGA microspheres for further development. PLGA is a well-known polymer, which has been widely applied in LAR formulations due to its biocompatibility, biodegradability, and favorable release kinetics. Veldoreotide has received orphan drug designation from the FDA and the European Medicines Agency.
KEVEYIS® is the first and only FDA-approved drug for the treatment of hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, a spectrum of rare and chronic genetic, neuromuscular disorders with autosomal dominant inheritance that cause recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis.1 As they age, some patients may experience permanent muscle weakness2. KEVEYIS® was approved by the FDA on August 7, 2015 and has received orphan drug exclusivity in the United States through August 7, 2022. Primary periodic paralysis is very rare, affecting approximately 4,000 to 5,000 diagnosed individuals in the U.S.3 The most common forms of the disease are hyperkalemic and hypokalemic Primary Periodic Paralysis, with an average time from onset of symptoms to correct diagnosis of 26 years.2,4
RECORLEV® and veldoreotide are not FDA-approved.
- Grieg SL. Dichlorphenamide: a review in primary periodic paralyses. Drugs. 2016;76:501- 507.
- Charles G, Zheng C, Lehmann-Horn F, Jurkatt-Rott, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260:2606-2613.
- Data on file. Feasterville-Trevose, PA: Strongbridge Biopharma; 2017.
- Cavel-Greant D, Lehmann-Horn F, Jurkat-Rott K. The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients. Acta Myol. 2012;31:126-133.