October 22, 2020         
FIBRA Prologis Announces Third Quarter 2020 Earnings Results   •   Black Women Leaders Host Statewide Call to Action in Support of Pro-Black Initiatives: Prop 15, Prop 16 and Prop 21   •   Morgan Stanley Launches Program to Provide Full Scholarships to Spelman College Students   •   America Will Rally for Afterschool Tomorrow as 21st Annual Lights On Afterschool Events Are Held Across the Country   •   Clo-Clo Vegan Foods New Full Line of Plant-Based Pizzas Adds a Novel Ingredient to the Recipe: Flavor!   •   shopDisney.com|Disney Store Unveil the Top 15 Toys for the 2020 Holiday Season   •   Tonal Announces Smart Flex, the Newest Dynamic Weight Mode in its Suite of Intelligent Weight Training Features   •   Consumers Demand Alcohol Cancer Warning Labels   •   Kilburn Live Teams Up With Dr. Seuss Enterprises For Brand New Holiday Experience Fit For Celebrating The 2020 Yuletide Season   •   Community Wellness Launches in California to Bring Remote Patient Monitoring Service to Older Adults with Cardiovascular Disease   •   Local Businesses Holding Relief Drive For Lake Charles Hurricane Survivors   •   Two Bit Circus Launches REMOTE: A Customizable, Play-From-Home Game Show, Hosted Live For Groups Of All Sizes   •   New COVID-19 Projections Show No Signs of Outbreak Abating in Midwest & Mountain States   •   PredictSpring Partners With Deciem Expanding Modern POS to Beauty Industry   •   InventHelp Inventor Develops Improved Sanitary Pad Product for Women (PND-5067)   •   Mini Golf, Monsters, Music and Fun Rolls into Ridge Hill in Yonkers, NY - Now Open   •   Coty Builds Digital & Luxury Strength With New Executive Appointments   •   Women 20 (W20) Challenges G20 Leaders to Close the Gap Between Imagination and Reality on Women’s Equality   •   Analytics Insight Announces 'The 10 Most Influential Women in Technology 2020'   •   FIBRA Prologis Receives Investment Grade Credit Rating
Bookmark and Share

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

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

PR Newswire

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/.

Media Contact

Jennifer Guinan | Sage Strategic Marketing | 610.410.8111 | Jennifer@sagestrat.com 

Cision View original content:http://www.prnewswire.com/news-releases/fda-grants-spirovant-sciences-orphan-drug-and-rare-pediatric-disease-designations-for-spiro-2101-for-treatment-of-cystic-fibrosis-301137781.html

SOURCE Spirovant

Back to top
| Back to home page

White House Live Stream
alsharpton Rev. Al Sharpton
9 to 11 am EST
jjackson Rev. Jesse Jackson
10 to noon CST


Sounds Make the News ®
Atlanta - WAOK-Urban
Berkley / San Francisco - KPFA-Progressive
Chicago - WVON-Urban
KJLH - Urban
Los Angeles - KJLH - Urban
WKDM-Mandarin Chinese
New York - WKDM-Mandarin Chinese
New York - WADO-Spanish
WBAI - Progressive
New York - WBAI - Progressive
Washington - WOL-Urban

Listen to United Natiosns News