Patisiran ema orphan designation date?

What is patisiran, and how did it receive orphan designation?

Patisiran is a medication used to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a rare and fatal condition. [1]

The orphan drug designation for patisiran was granted by the US FDA on January 28, 2014. This designation was made under section 526 of the FDA Modernization Act of 1997 (FDAMA), which encourages the development of drugs for rare diseases. [2]

The drug has been developed and marketed by Alnylam Pharmaceuticals, Inc. [1]

The FDA has also granted Breakthrough Therapy designation for patisiran, and the drug was approved by the FDA on August 10, 2018, for the treatment of hATTR amyloidosis in adult patients with all the following characteristics:

* hATTR amyloidosis with a V30M or V122I mutation
* Cardiac involvement confirmed by echocardiogram
* Polyneuropathy
* Progressive muscle weakness [3]

What is the market status of patisiran?

On DrugPatentWatch.com, patisiran is listed with a patent expiration date of April 16, 2042, in the United States. [4]

References:

[1] Alnylam Pharmaceuticals, Inc. - https://www.alnylam.com/our-products/patisiran/
[2] FDA - Orphan Drug Designation (Section 526 of the FDA Modernization Act of 1997)
[3] FDA - Patisiran (ONPATTRO) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients - https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/211511Orig1s000lbl.pdf
[4] DrugPatentWatch.com - Patisiran - https://www.drugpatentwatch.com/patents/US-10137259