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Regulatory Focus™ > News Articles > 2021 > 3 > FDA Approvals Roundup: Nulibry, Amondys 45, Pepaxto

FDA Approvals Roundup: Nulibry, Amondys 45, Pepaxto

Posted 03 March 2021 | By Renee Matthews 

FDA Approvals Roundup: Nulibry, Amondys 45, Pepaxto

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Nulibry okayed to cut mortality risk in babies with rare genetic disorder
Origin Biosciences’ Nulibry (fosdenopterin injection) has been approved for reducing the risk of death from molybdenum cofactor deficiency (MoCD) type A, a rare, genetic metabolic disorder marked by severe seizures. The condition presents soon after birth and most patients die in early childhood.
Approval of Nulibry was supported by efficacy data from three clinical trials compared with data from a natural history study, demonstrating that Nulibry reduced the risk of death by 82% in treated patients, compared with the untreated group. At three years, the probability of survival in 13 treated patients was 84%, compared with 55% for 18 untreated patients. Treatment with Nulibry also reduced urine concentrations of S-sulfocysteine, which builds up in MoCD and causes neurologic damage. That reduction was sustained with long-term treatment over 48 months.
This application was granted priority review, breakthrough therapy, and orphan drug designations. The sponsor also received a rare pediatric disease priority review voucher.
Amondys 45 approved as first therapy for rare Duchenne mutation
Sarepta’s Amondys 45 (casimersen injection) has been approved as a targeted, first therapy for Duchenne muscular dystrophy (DMD) patients who have a confirmed rare mutation.
DMD is a rare genetic disorder marked by progressive muscle deterioration and weakness. It is caused by a gene mutation that prevents formation of the muscle fiber protein dystrophin. In about 8% of DMD patients, the mutation is amenable to exon 45 skipping. By skipping exon 45, Amondys allows formation of a shortened but functional dystrophin protein.
Sarepta has two other approved DMD drugs that address exon 51- and exon 45-amenable forms of DMD.
Approval of Amondys 45 was based on findings from a double-blind, placebo-controlled study with 43 patients from the indicated population randomized 2:1 to receive the study drug or placebo. After 48 weeks, dystrophin protein levels were significantly higher from baseline in patients receiving Amondys 45, compared with those receiving placebo.
The findings did not show clinical benefit – for example, improved motor function – but the FDA said the demonstrated increase in dystrophin production could likely predict clinical benefit in patients with the mutation. The agency factored in the drug’s risks, the serious nature of the disease, and the lack of therapies for it, when deciding on the approval.
Amondys 45 was approved using the accelerated approval pathway, with continued approval depending on demonstration of clinical benefit in confirmatory trials. A clinical trial evaluating the drug’s safety and efficacy is currently underway. The application was granted fast track, priority review, and orphan drug designations.
Pepaxto greenlighted for triple-class refractory multiple myeloma
Oncopeptides’ Pepaxto (melphalan flufenamide) has been approved as a combination therapy with dexamethasone for relapsed/refractory multiple myeloma in previously treated patients who are refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.
The indication received an accelerated approval, based on response rate findings from the pivotal, phase 2, multicenter, single-arm HORIZON study in 157 patients. Of those patients, a subgroup of 97 had received previous treatment and were triple-class refractory. Overall response rate in the subgroup patients was 23.7% and median duration of response was 4.2 months. In all, 40 of the 97 (41%) patients had an aggressive and resistant disease associated with poor prognosis.
Pepaxto’s continued approval will depend on follow-up validation of clinical benefit in a confirmatory trial.
New indications
Humira expanded as treatment for active ulcerative colitis in children
AbbVie’s Humira(adalimumab) has been approved for treating moderate to severe active ulcerative colitis in children aged 5 years or older. The human anti-TNF monoclonal antibody is the only approved subcutaneous biologic treatment for this group of patients.
The extended indication was approved based on efficacy and safety findings from the pivotal phase 3, randomized, double-blind, multicenter ENVISION 1 study in patients aged 4 to 17 years patients with moderate to severe ulcerative colitis who received one of two dosages of Humira. After an 8-week induction period, 60% of patients taking the higher dosage attained clinical remission, compared with 43% of patients in the lower dosage group. At week 52, of the week 8 responders, 45% of higher-dosage patients attained remission, compared with 29% of lower-dosage patients and 33% of those on placebo. Humira’s safety profile in children was similar to that seen in adults with ulcerative colitis.

Humira was first approved in 2002 and is used to treat several types of autoimmune diseases, including rheumatoid arthritis, juvenile idiopathic arthritis, and Crohn’s disease.
Acetaminophen injection approved in multidose form
B. Braun’s acetaminophen injection has been approved in multidose packages of 1,000 mg in 100 mL and 500 mg in 50 mL doses, making it the only such injection available in multidose form.
Acetaminophen IV injection was first approved in 1951 and is used for managing mild to severe pain and reducing fever in adults and children.
Triumeq and Dovato get expanded indication for patients with HIV-1 and renal impairment
ViiV’s Triumeq (abacavir, dolutegravir, and lamivudine) has received an expanded indication for use in patients with HIV-1 infection and renal impairment with creatinine clearance greater than 30 to 49 mL/min. Approval of the supplemental new drug application also included a labeling update on Triumeq use with riociguat and during pregnancy.
Triumeq was first approved in 2014 for treating HIV-1 infection in adults and in children weighing 40 kg or more.
Viiv’s Dovato (dolutegravir and lamivudine), originally approved in 2019, also received a parallel approval for an expanded indication to include HIV-1 patients with the same moderate level of renal impairment.  


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Tags: FDA, US

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