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FDA Approves Boehringer’s Pediatric Oral Blood Thinner
Other recent FDA approvals include an edema drug for patients with heart failure, a systemic mastocytosis treatment, and a nasal antihistamine for allergic rhinitis.
FDA recently approved Boehringer Ingelheim Pharmaceuticals Pradaxa, the first oral blood thinning medication for children three months to less than 12 years old with venous thromboembolism.
Pradaxa was also approved to prevent recurrent clots among children who completed treatment for venous thromboembolism.
Additionally, the medication was approved in capsule form to treat blood clots in patients eight years of age and older who have been treated with a blood thinner for venous thromboembolism and to prevent recurrent clots in patients who completed prior treatment for at least five days.
“The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take,” Ann Farrell, MD, director of the division of non-malignant hematology in the FDA’s Center for Drug Evaluation and Research, said in the announcement.
“With today’s approval of Pradaxa, pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots,” Farrell continued.
In a study of 267 pediatric patients, 81 (45.8 percent) of the 177 individuals taking Pradaxa met the composite endpoint compared to 38 (42.4 percent) of the 90 patients who received standard of care.
The study’s primary endpoints were recurrence of blood clots, major and minor bleeding events, and death. Recurrence of blood clots occurred in just three patients (1.4 percent).
FDA Approves Edema Drug for Heart Failure Patients
Sarfez Pharmaceuticals recently announced that FDA approved its drug, Soaanz, to treat patients with heart failure who experience edema.
The once-a-day medication is an effective treatment option for patients who skip loop diuretic treatment due to concerns with excessive urination, as well as patients with chronic kidney disease.
Loop diuretics are used to treat volume overload caused by heart failure and chronic kidney disease, a Sarfez Pharmaceuticals spokesperson said. Overall, Soaanz provides a longer duration of peak effects without causing excessive urination.
Salim Shah, PhD, JD, founder and chairman of Sarfez Pharmaceuticals, stated that the company benefited from grant funding from the National Institute of Diabetes and Digestive and Kidney Diseases.
“We believe that for many of these patients who are aged 65 and older, the new treatment option now approved by the FDA offers tremendous benefits,” Shah continued. “We are thankful for that support, and we plan to transfer that benefit on to our patients by offering Soaanz at a reasonable price.”
FDA Approves Medication for Systemic Mastocytosis
Cambridge Massachusetts-based Blueprint Medicines recently announced that FDA has approved its systemic mastocytosis treatment (Advanced SM), Ayvakit.
This is the first target therapy approved for adult patients with systemic mastocytosis, including aggressive SM, SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
The therapy is designed to potently inhibit D816V mutant KIT, the central driver of the disease, a Blueprint Medicines spokesperson explained.
“The approval of AYVAKIT for advanced systemic mastocytosis – the fourth FDA approval across our portfolio in 18 months – culminates nearly a decade of hard work, from our scientists in the laboratory and clinical team conducting trials, to our commercial organization who will now bring AYVAKIT to patients,” Jeff Albers, chief executive officer of Blueprint Medicines, said in the announcement.
FDA granted full approval to Ayvakit based on data from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial.
In the trials, researchers found a resolution of at least one finding of non-hematologic and hematologic organ damage and 50 percent or greater reductions in biomarker response, mast cell burden, and serum tryptase.
In 53 patients, the overall response rate nearly one year after treatment was 57 percent. And the median duration of response was 38.3 months.
Notably, Ayvakit is not recommended for the treatment of patients with advanced SM with low platelet counts.
FDA Approves Nasal Antihistamine for Allergic Rhinitis
FDA recently approved Bayer’s Astepro, an azelastine hydrochloride nasal spray for seasonal and perennial allergic rhinitis.
The nasal spray, which was approved for nonprescription use through a process called partial prescription and nonprescription switch, is intended for adults and children six years of age and older.
According to FDA, for a drug to switch from prescription to nonprescription status, the drug’s data must show that the drug is safe and effective for use in self-medication as directed in labeling.
“Seasonal and perennial allergies affect millions of Americans every year, causing them to experience symptoms of nasal congestion, runny nose, sneezing and more,” Theresa M. Michele, MD, director of the office of nonprescription drugs in the FDA’s Center for Drug Evaluation and Research, said in the announcement.
“Today’s approval provides individuals an option for a safe and effective nasal antihistamine without requiring the assistance of a healthcare provider,” Michele continued.
