(Corrects to remove the word 'genetic' from headline. An
earlier version of the story was corrected to clarify that loss
of exclusivity year is "2030", not "2028", in paragraph 7)
March 10 (Reuters) - The U.S. Food and Drug
Administration approved Acadia Pharmaceuticals Inc's drug for the treatment of Rett syndrome, a genetic brain
disorder, the company said on Friday, making it the first
approved drug for the condition.
The U.S. health regulator's decision allows use of the
trofinetide, to be sold under the brand name Daybue, in adult
and pediatric patients two years of age and older and comes with
a warning of diarrhea and weight loss.
The approval comes months after the FDA declined to approve
expanded use of Acadia's drug Nuplazid to treat psychosis
related to Alzheimer's disease. Analysts have said approval of
Daybue would help drive growth for the company in the near term.
"We have put a lot of planning into potential
commercialization of trofinetide, including resources for
patients to access the drug," said Acadia senior executive
Kathie Bishop ahead of the approval.
Acadia said it plans to make the drug available to patients
by the end of April. It did not disclose details of the price.
Before the approval, David Hoang SMBC Nikko Securities
analyst estimated a list price at launch of $450,000 annually.
He forecast peak U.S. trofinetide sales of $487.2 million by
2035. RBC Capital Markets analyst Gregory Renza, also writing
before the approval, predicted peak U.S. sales to exceed $500
million by 2032 and an average annual launch price of about
$425,000.
Acadia forecasts sales of Nuplazid - its only drug on the
market - of between $520 and $550 million this year, above
analysts' median expectations of $532.8 million, according to
Refinitiv data. With the drugmaker facing a loss of exclusivity
for Nuplazid in 2030, investors have pinned their hopes on a
successful trofinetide launch.
After the FDA declined to approve the expanded use of
Nuplazid, Acadia said it would not pursue that indication for
Nuplazid further. The drugmaker plans to focus its resources on
late-stage development of Nuplazid to treat symptoms of
schizophrenia and early-stage development of another candidate,
ACP-204, for Alzheimer's-related psychosis.
Rett's syndrome is a rare neurodevelopmental condition that
occurs primarily in girls. According to government estimates, it
affects fewer than 50,000 people in the U.S.
Shares of the California-based company closed 0.68% lower on
Friday.
Acadia's drug acts as an artificial form of the insulin-like
growth factor IGF-1 and helps reduces inflammation in the
nervous tissue as well as aid in the transmission of nerve
impulses.
Acadia's application for marketing approval was based on
data from a late-stage study in which treatment with the drug
showed improvement in core symptoms of the disease compared to a
placebo. Improvement of symptoms was measured according to the
assessment scales Rett Syndrome Behaviour Questionnaire and the
Clinical Global Impression of Improvement.
The company had licensed the drug for $10 million up front
payment from Australian drugmaker Neuron Pharmaceuticals for development and sale in North America in 2018.
(Reporting by Bhanvi Satija, Nandhini Srinivasan and Anirudh
Saligrama in Bengaluru; Editing by Shailesh Kuber and William
Mallard)
9873062788;))
