Blueprint Medicines announces FDA acceptance of AYVAKIT® supplemental NDA for indolent systemic mastocytosis treatment. FDA grants priority review with May 22, 2023 action date under PDUFA. Results from the PIONEER trial, the largest randomized placebo-controlled study in indolent SM, supports application. AYVAKIT designed to selectively inhibit D816V mutant KIT-causing SM and previously granted breakthrough therapy designation by FDA.

Publish Date: 23-01-2023 Source: Blueprint Medicines

The most severe Adrenoleukodystrophy (ALD), a rare X-linked metabolic illness, is Cerebral Adrenoleukodystrophy (CALD). Between 40 and 72 percent of boys diagnosed with ALD will proceed to cerebral ALD, usually between 3 and 12. In most untreated patients, cerebral ALD is characterized by a quickly progressive neurologic decline leading to significant neurologic function loss and death. It’s a devastating condition for affected boys and their families, with severe symptom burden and rapid deterioration in neurologic function if left untreated. This is why assembling a care team to monitor your condition as soon as possible is critical.

• Based on literature research, epidemiology study, and Thelansis, the diagnosed prevalence of Adrenoleukodystrophy is approximate ~78,000 cases in 8 major markets for 2020.

However, the current Indolent Systemic Mastocytosis treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (PA101, Masitinib, etc.) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Indolent Systemic Mastocytosis treatment. The key companies in the advanced development stage are Patara Pharma, AB Science, etc.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Indolent Systemic Mastocytosis to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2022 to 2032.Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

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