The FDA rejected Mesoblast’s NDA for remestemcel-L as a treatment for children with steroid-resistant graft-versus-host disease and requested data from at least one additional clinical trial. The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 9-1 to recommend approval of remestemcel-L in August 2020. Because of the overwhelming ODAC vote, Mesoblast plans to appeal the decision.
Protalix BioTherapeutics’ pegunigalsidase alpha is available through an expanded access program (NCT04552691) for patients with Fabry disease that have exhausted their treatment options.
The FDA has granted Italfarmaco’s givinostat a Rare Pediatric Disease designation for the treatment of Duchenne muscular dystrophy.
The FDA allowed the IGNITE-DMD trial to continue after Solid Biosciences improved its manufacturing and enrollment procedures for investigational Duchenne muscular dystrophy gene therapy, SGT-001.
The FDA has granted Pfizer’s minidystrophin gene therapy, PF-06939926, Fast Track status for the treatment of Duchenne muscular dystrophy.
Kadmon completed a rolling NDA for belumosudil to treat chronic graft-versus-host disease.
Announced Research Updates
Sarepta announced 18-month interim results from three patients enrolled in a Phase I/IIa, open-label trial, where treatment with a low dose of SRP-9003 improved the North Star Assessment for Dysferlinopathy (NSAD) by 5.7 points in patients with limb-girdle muscular dystrophy. Patients also saw an improvement in time to rise, four-stair climb, 100-meter walk test and 10-meter walk test. Sarepta also announced 6-month results from three patients that received a high dose of SRP-9003 from the same trial, where a high dose of SRP-9003 demonstrated an improvement in NSAD, time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test. At six-months, the NSAD score had improved 3.7 points with the high dose of SRP-9003 compared to a 3 point improvement with the low dose.
Alnylam announced that in the 6-month, 18 patient, Phase III, open-label, ILLUMINATE-B trial, treatment with lumasiran decreased spot urinary oxalate:creatinine ratio compared to placebo in children < six years with primary hyperoxaluria type 1.
Published Research Updates
In two 52-week, Phase III trials that enrolled a total of 1,771 patients, (LUSTER 1 and LUSTER 2), treatment with fevipiprant did not reduce moderate-to-severe exacerbation compared to placebo in patients with inadequately controlled moderate-to-severe asthma.
In the 71 patient, Phase I, GARNET trial, treatment with dostarlimab resulted in a 42.3% objective response rate in patients with deficient mismatch mutation repair endometrial cancers after prior platinum-based chemotherapy.
After 16-weeks in the 52-week, 802 patient, Phase III ECZTRA 1 trial, 15.8% of patients treated with tralokinumab achieved an Investigator Global Assessment (IGA) score of clear (0) or almost clear (1) skin and 25% had at least a 75% or > improvement in their Eczema Area and Severity Index (EASI) score compared to 7.1% and 12.7% with placebo in patients with moderate-to-severe atopic dermatitis with an inadequate response to topical treatments.
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