RARE Daily

Rhythm Pharmaceuticals Sells Priority Review Voucher to Alexion for $100M

January 6, 2021

Rare Daily Staff

Rhythm Pharmaceuticals, a biotech focused on developing therapies to treat rare genetic diseases of obesity, said it has entered into a definitive agreement to sell its Rare Disease Priority Review Voucher for $100 million.

Although Rhythm did not publicly disclose to whom it would sell the PRV, SEC documents showed that the sale is to Alexion Pharmaceuticals, a leader in complement-mediated rare disease therapies that is soon to be acquired by AstraZeneca for $39 billion.

The PRV was granted to Rhythm by The U.S. Food and Drug Administration granted the PRV to Rhythm with the approval of Imcivree (setmelanotide) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing.

“Rhythm is focused on transforming the care of people living with rare genetic diseases of obesity,” said David Meeker, chair, president, and CEO of Rhythm. “The non-dilutive capital from the sale of our PRV provides an important source of additional funding to advance the continued development of setmelanotide as a precision medicine for people whose severe obesity and insatiable hunger that may be caused by genetic variants associated with the melanocortin-4 (MC4R) receptor pathway.”

According to the agreement, Rhythm will receive a payment of $100 million upon the closing of the transaction, which is subject to customary closing conditions and is expected to occur following expiration of the applicable U.S. antitrust clearance requirements.

The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. A PRV may be issued to the sponsor of a rare pediatric disease product application and would entitle the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval. The sponsor receives the PRV upon approval of the rare pediatric disease product application and it can be sold without limitation, subject to applicable FDA requirements for filing and use.

According to the current statutory sunset provisions, after December 11, 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by December 11, 2020. After December 11, 2022, FDA may not award any rare pediatric disease priority review vouchers.

Photo: David Meeker, chair, president, and CEO of Rhythm

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