Rocket Pharma is getting ready to light up national landmarks on Thursday as part of its annual rare disease awareness effort, but this year the event is even more meaningful as Rocket awaits an FDA decision on its first drug candidate.

Rocket’s gene therapy candidate, Kresladi, to treat severe leukocyte adhesion deficiency-I (LAD-I) is being considered under priority review with a June 30 decision date. Rocket also plans to submit another biologics license application (BLA) in the first half of the year for RP-L102 treatment for fanconi anemia.

“For Rocket we focused from day one starting the company to do something for the rare disease patient and community before we have therapies in the clinic,” said Rocket president and chief operating officer Kinnari Patel.

Along with the Empire State Building in New York, where Rocket has offices, the FDA headquarters, Niagara Falls, and NASDAQ are among the more than 100 places around the world that will light up in the green, blue, pink and purple colors that represent rare disease. Rocket first began the illumination campaign in 2019, although the National Organization for Rare Disease (NORD) took over more recently.

Meanwhile, Rocket also is planning a day of panel discussions and events on Thursday, as well as a media push, to draw attention to rare diseases, which in total affect 10% of people in the US. Themes for the day center on patient support, patient advocacy and genetic testing, Patel said.

Source:
https://endpts.com/rocket-pharma-lights-up-buildings-again-for-rare-disease-day-as-it-awaits-first-fda-decision/
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