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 vertical, therapeutically-aligned rare disease franchises. Strongbridge's rare endocrine franchise includes MACRILEN™ (macimorelin), the first and only FDA-approved oral drug indicated for the diagnosis of adult growth hormone deficiency, RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies for the treatment of endogenous Cushing's syndrome, and veldoreotide extended release, a pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. MACRILEN has orphan drug exclusivity in the United States, and both RECORLEV and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. 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. Significant unmet needs remain in the diagnosis and treatment of these rare diseases.
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
MACRILEN™ (macimorelin), an oral growth hormone secretagogue receptor agonist, is the first and only FDA-approved oral drug indicated for the diagnosis of Adult Growth Hormone Deficiency.1 MACRILEN was approved by the FDA on December 20, 2017, has been granted orphan drug exclusivity in the United States, and has patents with expiration dates through late 2027. Adult growth hormone deficiency (AGHD) affects more than 50,000 adults in the U.S. Growth hormone is a protein synthesized, stored, and secreted by the pituitary gland. Adults need growth hormone to maintain healthy tissue and organ function. Adult growth hormone deficiency is typically caused by injury/insult to the pituitary and leads to metabolic, cardiovascular, musculoskeletal, and mental health issues. It is treatable with recombinant exogenous growth hormone.2-4
RECORLEV, a cortisol synthesis inhibitor, is currently being studied in Phase 3 clinical studies for the treatment of endogenous Cushing's syndrome. SONICS, the first global Phase 3 study is fully enrolled and supported by LOGICS, a second global Phase 3 study designed to supplement the long-term efficacy and safety data from the SONICS study in a randomized, double-blind, placebo-controlled study. Endogenous Cushing’s syndrome is a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. People with uncontrolled disease are seriously ill and have a 2- to 4-fold higher mortality rate than age- and gender-matched controls, mainly due to metabolic and cardiovascular complications. RECORLEV has received orphan drug designation from the FDA and the European Medicines Agency. The Company is developing RECORLEV, a single enantiomer of ketoconazole, as a new chemical entity, or NCE, under the FDA 505(b)(2) regulatory pathway, and intends to reference the FDA’s prior conclusions of safety for ketoconazole.
Veldoreotide extended release, a pre-clinical next-generation somatostatin analog, is being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Veldoreotide may provide the acromegaly community with a new treatment option offering a unique clinical profile and delivery system. 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.
The safety and efficacy of RECORLEV and veldoreotide have not been established.
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.5 As they age, some patients may experience permanent muscle weakness6. 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.7 The most common forms of the disease are hyperkalemic and hypokalemic Primary Periodic Paralysis, and time between symptom onset and diagnosis can take more than 20 years.6,8
- MACRILEN Prescribing Information. 2018.
- Monson JP, Brooke AM, Akker S. Adult Growth Hormone Deficiency. [Updated 2015 May 19]. In: De Groot LJ, Chrousos G, Dungan K, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000-.
- Devesa, Jesús, Cristina Almengló, and Pablo Devesa. “Multiple Effects of Growth Hormone in the Body: Is It Really the Hormone for Growth?” Clinical Medicine Insights. Endocrinology and Diabetes 9 (2016): 47–71. PMC. Web. 16 Jan. 2018.
- Gupta, Vishal. “Adult Growth Hormone Deficiency.” Indian Journal of Endocrinology and Metabolism 15.Suppl3 (2011): S197–S202. PMC. Web. 16 Jan. 2018.
- 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.