BioBlast Pharma (NASDAQ:ORPN) of Tel Aviv has been granted Orphan Drug status by the U.S. Food & Drug Administration (FDA) for its product candidate Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (also known as SCA3 or Machado-Joseph disease. This is the second indication for which BioBlast’s Cabaletta has received this designation.
The Orphan Drug Designation program provides orphan status to novel drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
If approved, the benefits of Orphan Drug status include:
- 7-year marketing exclusivity
- Tax credits (up to 50% of clinical development costs)
- Ability to apply for annual grant funding
- Exemption/Waiver of Prescription Drug User Fee Act (PDUFA) application (filing) fees
- OOPD (Office of Orphan Product Development) assistance during the development process
Cabaletta is “a chemical chaperone that protects against pathological processes in cells. It has been shown to prevent pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation. Cabaletta has demonstrated efficacy in preclinical cells and animal models of SCA3 and other PolyA/PolyQ diseases, including Occulopharyngeal Muscular Dystrophy (OPMD) and Spino bulbar cerebellar ataxia (SBMA, or Kennedy’s disease). BioBlast plans to make clinical progress in each of these indications in 2015.”
Dalia Megiddo, BioBlast CEO, made the following statement:
This orphan drug designation lays the foundation for what we believe are very favorable conditions to continue to pursue our clinical plans and reflects our commitment to those patients suffering from rare diseases for which there are few or no therapeutic options. It is yet another step on BioBlast’s route to create and capture value from our research and clinical work. We currently are conducting a phase 2 clinical trial to test the efficacy of Cabaletta in SCA3 and plan to start our SCA3 pivotal study in 2015.
The announcement comes just two months after the company celebrated its IPO by visiting the NASDAQ Marketsite in Times Square and ringing the NASDAQ opening bell. Soon after, BioBlast announced the initiation of its Canadian OPMD Clinical Study Center at McGill University in Montreal.
Spinocerebellar Ataxia Type 3
According to the U.S. National Library of Medicine, Spinocerebellar ataxia type 3 (SCA3) is characterized by progressive problems with movement. People with this condition initially experience problems with coordination and balance (ataxia). Other early signs and symptoms of SCA3 include speech difficulties, uncontrolled muscle tensing, muscle stiffness, rigidity, tremors, bulging eyes, and double vision. They may experience sleep disorders such as restless leg syndrome or REM sleep behavior disorder. These sleep disorders tend to leave affected individuals feeling tired during the day.
Over time, individuals with SCA3 may develop loss of sensation and weakness in the limbs, muscle cramps, muscle twitches, and swallowing difficulties. Individuals with SCA3 may have problems with memory, planning, and problem solving.
Signs and symptoms of the disorder typically begin in mid-adulthood but can appear anytime from childhood to late adulthood. People with SCA3 eventually require wheelchair assistance. They usually survive 10 to 20 years after symptoms first appear.
BioBlast Pharma is a clinical-stage biotechnology company that develops therapies for patients with rare and ultra-rare genetic diseases. Founded in 2012, the company is building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms potentially offer solutions for several diseases that share the same biological pathology. For more information, visit www.bioblast-pharma.com.