FDA Approvals Roundup: Pradaxa, Ayvakit, Astepro

| 23 June 2021 | By Renee Matthews 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New indications
Pradaxa gets new indication as oral therapy for VTE in children
Boehringer Ingelheim’s Pradaxa (dabigatran etexilate oral pellets) has received an expanded indication for treating venous thromboembolism (VTE) in children aged 3 months to younger than 12 years after they have received blood thinner by injection for at least five days. The oral pellet form of Pradaxa was also approved for prevention of recurrent clots in patients in this age population who completed treatment for their first VTE. This is the first FDA approval of an oral anticoagulant for children.
A capsule form of the drug was also approved for patients aged 8 years or older with VTE for the same indications. The only other approved blood thinning medication for children is given by injection.
Approval of Pradaxa was based on safety and efficacy findings from two open-label studies. In the first study, the drug’s safety and efficacy were evaluated in 267 patients younger than 18 years from the indicated population. They were randomized to receive Pradaxa or standard of care. The two groups were evaluated and compared based on the number of patients in each who met a composite endpoint. Meeting the composite endpoint meant a patient had not died from a blood clot, the blood clots had completely resolved, and there were no additional blood clots. The findings showed that 45.8% of patients (81/177) who received Pradaxa met the composite endpoint, compared with 42.2% of patients (38/90) who received standard of care.
The second study evaluated Pradaxa’s safety in preventing recurrent blood clots in 214 patients younger than 18 years and with a history of blood clots. The primary endpoints of this single-arm study were recurrence of blood clots, major and minor bleeding events, and overall and clot-related death. Findings showed the drug’s safety with long-term use was similar to that in the aforementioned study. In all, 1.4% of patients (3/214) had a recurrence of blood clots, which was comparable with the standard-of-care therapy.
Pradaxa received a priority review designation for this indication.
Pradaxa was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with nonvalvular atrial fibrillation.
Ayvakit use expanded to include advanced systemic mastocytosis
Blueprint Medicines’ Ayvakit (avapritinib) has received an expanded indication for use in adults with advanced systemic mastocytosis.
Approval of Ayvakit was based on efficacy findings from the multicenter, single-arm, open-label EXPLORER and PATHFINDER clinical trials in 53 patients from the indicated population. The combined overall response rate in all evaluable patients in both trials was 57%, with 28% complete remissions, and 28% partial remissions. The median duration of response was 38.3 months and the median time to response was 2.1 months.
This application was granted priority review and breakthrough and orphan drug designations.
Ayvakit was originally approved in 2020 for treating adults with unresectable or metastatic gastrointestinal stromal tumor with certain mutations.
Astepro approved as OTC nasal antihistamine for seasonal allergies
Bayer’s Astepro (azelastine hydrochloride nasal spray, 0.15%) has been approved as an over-the-counter therapy for seasonal and perennial allergies in patients aged 6 years or older.
The process is known as a partial prescription to nonprescription switch because the perennial allergy indication for children aged 6 months to 6 years and the seasonal allergy indication for children aged 2 to 6 years will remain prescription based.
For a drug to make this switch, the sponsor must provide data showing the therapy is safe and effective for use in self-medication. The manufacturer must also show consumers can understand how to use the drug safely and effectively without the supervision of a healthcare professional.
Astepro was originally approved in 2008 as a new formulation of Astelin.



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Tags: FDA, US

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