Sevuparin receives Rare Pediatric Disease Designation from FDA for the treatment of children with sickle cell disease
STOCKHOLM, SWEDEN – 18 April 2018: Modus Therapeutics AB, a company developing innovative treatments in high unmet medical need disease areas with a focus on sickle cell disease (SCD), has announced that sevuparin for the treatment of SCD was granted rare pediatric disease designation by the US Food & Drug Administration (FDA).
Sickle cell disease, an inherited blood disorder characterized by severe pain leading to organ damage, affects between 90,000-100,000 individuals in the U.S. Progressive organ damage limits the life expectancy of patients with SCD and lifetime medical care costs can exceed $1M per patient with an estimated $1B spent annually in the U.S.
Sevuparin is an innovative, proprietary polysaccharide drug with anti-adhesive, anti-aggregate and anti-inflammatory effects due to its multimodal mechanism of action. Sevuparin has the potential to restore blood flow and prevent further microvascular obstructions in children and adults with SCD. Children with SCD suffer from some of the most serious and life-threatening consequences of the disease including stroke, splenic sequestration and aplastic anemia. Mortality is a significant concern in children globally, with 92% of newborns dying in the first few years of life due to lack of early diagnosis.
Ellen K Donnelly, PhD, CEO of Modus Therapeutics, said: “We are very pleased with the FDA’s decision to grant a rare pediatric disease designation to sevuparin for SCD. The potential for Modus Therapeutics to be granted a Rare Disease Priority Review Voucher upon marketing approval of sevuparin could be an additional value driver for our shareholders.”
Created in 2014 by Congress, the Rare Pediatric Disease Priority Review Voucher Program provides incentive for the development of treatments for rare pediatric diseases. A drug qualifies for this category if the entire prevalence of the disease in the US is below 200,000 and the serious or life-threatening manifestations of the disease primarily affect individuals from birth to age 18.
If a drug candidate with a rare pediatric disease designation receives marketing approval in the US, the FDA may issue a Priority Review Voucher to the sponsor company. This voucher can be redeemed to provide Priority Review of a subsequent marketing application for a different product from Modus Therapeutics or another company.
For further information, please contact:
Ellen K Donnelly, CEO, Modus Therapeutics:
Tel: +46 70 246 7554
David Dible/ Pip Batty, Citigate Dewe Rogerson
Tel: +44 20 7638 9571
FOR THE EDITORS
About Modus Therapeutics AB
Modus Therapeutics is a Swedish biotech company developing sevuparin for diseases with high unmet medical need with a focus on sickle cell disease (SCD) and Malaria. Sevuparin is currently being tested in a Phase II clinical study for SCD – a painful, inherited blood disorder affecting millions of people around the globe. SCD patients’ blood cells form a sickled shape, which makes blood flow to vital organs difficult, causing severe pain and even premature death. Sevuparin has the potential to improve the SCD patients’ blood flow reducing their pain and the amount of time they will need to spend in hospital. Modus plans to develop an administration form of sevuparin that the patient can self-administer allowing them to live a more normal life by preventing the painful episodes requiring hospital care.
Modus is predominantly owned by KDev Investments AB, part of Karolinska Development AB (Nasdaq Stockholm: KDEV) and Rosetta Capital. Other larger owners are The Foundation for Baltic and European Studies (Östersjöstiftelsen) and Praktikerinvest AB. For more information, please visit www.modustx.com
Sevuparin is an innovative, proprietary polysaccharide drug with anti-adhesive, anti-aggregate and anti-inflammatory effects due to its multimodal mechanism of action. Sevuparin has the potential to restore blood flow and prevent further microvascular obstructions in a number of diseases including SCD and malaria. In SCD, the anti-adhesive effects of sevuparin could thereby offer treatment of the underlying cause of vaso-occlusive crisis (VOC) in SCD patients, with earlier pain relief, shorter hospital stays, reduced need of opioids and improved quality of life. Modus is currently enrolling patients in a Phase II study with the aim to complete the study in late 2018.
About sickle cell disease
Sickle cell disease (SCD) is a painful, inherited blood disorder affecting millions of people around the globe and the most common inherited blood disorder in the U.S. affecting between 90,000-100,000 subjects, with medical care costs amounting to more than $1 billion. In Europe it is estimated that there are 35,000-40,000 SCD patients, with an even higher number in the Middle East and North Africa regions, with over 850,000 SCD patients.
There is currently no pharmaceutical product available that targets the underlying cause of VOCs that affect SCD patients. Current therapies are predominantly strong intravenous pain medications and SCD patients often must be hospitalized to be treated.