New Jer­sey’s Rock­et Phar­ma­ceu­ti­cals hit a ma­jor road­block for its AAV-de­liv­ered gene ther­a­py for heart fail­ure in May af­ter the FDA came ask­ing for more de­tails on tar­get pa­tients. Three months down the road, Rock­et is clear for liftoff once again.

The FDA on Mon­day lift­ed its clin­i­cal hold on Rock­et’s Phase I tri­al for gene ther­a­py can­di­date RP-A501 for Danon dis­ease, an ul­tra-rare ge­net­ic dis­or­der that can lead to heart fail­ure in chil­dren and young adults, the com­pa­ny said in a re­lease.

It’s been a lit­tle more than three-month wait for Rock­et af­ter the agency ground­ed the tri­al, ask­ing for a re­vised pro­to­col on “pa­tient se­lec­tion and man­age­ment.”

On Mon­day, Rock­et said it had ad­dressed those is­sues and was ready to move for­ward, cit­ing no new safe­ty red flags churned up dur­ing the agency’s re­view. The com­pa­ny ex­pects to re­sume dos­ing in the adult co­hort “as soon as pos­si­ble” and is gear­ing up to be­gin en­roll­ment for a pe­di­atric co­hort as well, Rock­et said.

Un­like the rest of its pipeline, which us­es lentivi­ral vec­tors to trans­port its gene ther­a­pies, RP-A501 is Rock­et’s on­ly can­di­date that us­es AAV9 for de­liv­ery, a vec­tor that has re­ceived in­tense scruti­ny af­ter a wave of safe­ty flags in re­cent years. The ther­a­py us­es that vec­tor to trans­port a func­tion­al ver­sion of lyso­some-as­so­ci­at­ed mem­brane pro­tein 2 (LAMP-2) in­to pa­tients’ cells — the mu­tat­ed form of the pro­tein caus­es a buildup of au­tophago­somes and glyco­gen in the heart and oth­er tis­sues that can cause heart fail­ure and death, par­tic­u­lar­ly in boys and young men.

Rock­et be­lieves there are some­where be­tween 15,000 and 30,000 pa­tients with Danon in the US and Eu­rope. The drug would be­come the first gene ther­a­py ap­proved for mono­genic heart fail­ure, Rock­et said.

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

The White House’s Office of Science and Technology Policy (OSTP) last year sought to find ways to better coordinate large-scale clinical trials in the US — as the UK lead by example during the pandemic — especially for these emergency clinical trials.

The lobbying group PhRMA Tuesday called for more clinical trial diversity in underserved areas, including by making participation less of a burden, and expanding eligibility criteria when appropriate.