FDA Grants Spirovant Sciences Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 for Treatment of Cystic Fibrosis

PHILADELPHIA, Sept. 24, 2020 /PRNewswire/ — Spirovant Sciences, a gene therapy company developing treatments and cures for genetic lung diseases including cystic fibrosis, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease Designations to its lead product candidate, SPIRO-2101, for the treatment of cystic fibrosis.

SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator. SPIRO-2101 contains an evolved AAV capsid engineered to have high tropism to human airway epithelia. This treatment is designed for these patients who currently have no approved modulator therapies.

“Cystic fibrosis is an incredibly debilitating and devastating disease,” said Joan Lau, Ph.D., Chief Executive Officer of Spirovant Sciences. “Receiving Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 underscores the urgency for a treatment option for these patients, particularly those with very rare and more severe types of cystic fibrosis, the nonsense mutation subtype. With these important regulatory milestones, combined with our amazing and growing team, we are well positioned to advance SPIRO-2101 into the clinic and offer hope to these patients.”

The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

Rare Pediatric Disease Designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. Under the Rare Pediatric Disease program, a sponsor that receives approval for a drug or biologic for a “rare pediatric disease” may qualify for a priority review voucher that may be sold or transferred.

About Cystic Fibrosis
Cystic fibrosis is a life-threatening, autosomal recessive genetic disease with no cure. It is caused by mutations of the gene encoding the CFTR, a channel that conducts chloride and bicarbonate ions across epithelial cell membranes. Derangement of CFTR interferes with mucociliary transport and antibacterial activity in the airways and gut, which leads to a buildup of mucus and susceptibility to airway infections that is ultimately lethal.  Cystic fibrosis is primarily characterized by progressive airway infection and inflammation, and, in most individuals, death from respiratory failure.

There are an estimated 75,000 cystic fibrosis patients worldwide.  The standard of care for cystic fibrosis includes therapies that address symptoms of the disease, such as bronchodilators, mucolytics, and antibiotics, and those that attempt to modulate the activity of defective CFTR, such as correctors of CFTR trafficking or potentiators of impaired ion channel activity. None of the current treatments prevent continued disease progression and many have no efficacy for individuals with class 1 (nonsense) mutations or no CFTR protein at all or for those do not tolerate a modulator, which comprise approximately 10% of the total cystic fibrosis patient population.

About Spirovant Sciences, Inc.
Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other genetic lung diseases. The company’s current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis. Spirovant is advancing programs for cystic fibrosis with both AAV and lentivirus vectors. Spirovant is a wholly owned subsidiary of Sumitovant Biopharma Ltd., which is itself a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd.  Spirovant is located in Philadelphia, PA. More information is available at https://www.spirovant.com/.

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant and Urovant, and wholly owns Enzyvant, Spirovant and Altavant. Sumitovant’s pipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need. For further information about Sumitovant please visit https://www.sumitovant.com/

About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com/.