The clock is ticking. If you or someone you love is diagnosed with a rare disease like hypoplasminogenemia, days — often even hours — can mean the difference between life and death.
So when it comes to speeding up the process for new treatments’ approval by the U.S. Food and Drug Administration (FDA), a few months can literally mean a lifetime to people desperately waiting for relief. And when the FDA grants Rare Pediatric Disease Designation to a promising therapy, families rejoice.
According to a press release issued by Canadian biopharmaceutical corporation Prometic, on August 29, the FDA granted a Rare Pediatric Disease Designation to Prometic’s Ryplazimä, a plasminogen replacement therapy for the treatment of patients with congenital plasminogen deficiency.
In addition to the Rare Pediatric Disease Designation, Ryplazimä has already been granted Orphan Drug and Fast Track Designation by the FDA. That means what could have taken 10 months or more for a standard FDA review, will take only six months. And four months can mean a lot to a child with hypoplasminogenemia, also called type 1 plasminogen deficiency.
Hypoplasminogenemia is a genetic condition that causes inflamed growths on the eyes, mouth, ears, throat and other mucous membranes. The most commonly affected area is the conjunctiva, where the mucous membranes protect the whites of the eye and eyelids. But it can sometimes lead to very serious complications, according to National Organization for Rare Diseases (NORD).
Growths in the respiratory tract may cause recurrent pneumonia and obstruction of the airways. Growths in the gastrointestinal tract may result in ulcers. Growths in the windpipe can potentially cause life-threatening obstruction, particularly in small children. And some children with congenital plasminogen deficiency can develop a build-up of fluid in the brain.
The exact prevalence of the disorder is unknown, but one estimate places the incidence at 1.6 people per 1,000,000 in the general population.
The FDA grants Rare Pediatric Disease Designation for serious or life-threatening diseases in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents. If approved, Prometic’s plasminogen replacement therapy will be eligible to receive a rare pediatric disease priority review voucher.
“The pediatric designation also provides an important incentive for Prometic to continue developing therapies addressing unmet medical needs for children with rare diseases,” said Mr. Pierre Laurin, President and Chief Executive Officer of Prometic. “We look forward to continuing to work closely with the FDA to bringing this innovative therapy to patients suffering from plasminogen deficiency.”
