FDA Approvals Roundup: Scemblix, Vuity, Cortrophin Gel

RoundupsRoundups | 03 November 2021 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Scemblix okayed for chronic myeloid leukemia with Philadelphia mutation
Novartis’s Scemblix (asciminib) has been granted accelerated approval as a therapy for adults with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML in CP) who have received previous treatment with two or more tyrosine kinase inhibitors (TKIs), as well as approval for use in adults with Ph+ CML in CP who have the T315I mutation.
The accelerated approval of Scemblix, a kinase inhibitor, for TKI-resistant patients was based on findings from the multicenter, randomized, active-controlled, open-label ASCEMBL clinical trial in 233 patients from the indicated population randomized 2:1 to receive Scemblix or bosutinib until unacceptable toxicity or treatment failure occurred. At 24 weeks, the major molecular response rate (MMR) was 25% in patients receiving Scemblix and 13% in bosutinib patients. Median duration of follow-up was 20 months, and median duration of MMR has not yet been reached.
Approval of the therapy for patients with Ph+ CML in CP with the T315I mutation was based on findings from the multicenter, open-label CABL001X2101 clinical trial in 45 patients with the T315I mutation. The patients received twice-daily Scemblix until unacceptable toxicity or treatment failure. The main efficacy outcome measure was MMR. In all, 42% of patients achieved MMR by 24 weeks, and 49% achieved MMR by 96. Median duration of treatment was 108 weeks.
The review for this application used the real-time oncology review pilot program and the assessment aid. This application was granted priority review and breakthrough, fast track, and orphan drug designations.
Vuity eye drops approved for age-related blurry near vision
AbbVie’s Vuity (pilocarpine HCl ophthalmic solution, 1.25%) has been approved for treating presbyopia, or age-related blurry near vision, in adults.
Approval of Vuity, a cholinergic muscarinic receptor agonist, was based on findings from the phase 3 randomized, double-masked, vehicle-controlled GEMINI 1 and GEMINI 2 clinical studies in a total of 750 participants from the indicated population. Participants were randomized 1:1 to self-administer 1 drop of Vuity or placebo once daily in each eye for 30 days using the pHast delivery technology that allows Vuity to reach the pH of the physiologic tear film rapidly.
Pilocarpine, Vuity’s active ingredient, causes temporary miosis, or constriction of the pupil, improving near vision without sacrificing far vision.
In both studies, participants receiving Vuity had statistically significant improvements in near vision in low-light conditions and without a loss of distance vision at hour 3 of day 30 in the intent-to-treat population. In GEMINI 1, 31% of participants showed improvements, compared with 8% in the placebo group; in GEMINI 2, those proportions were 26% and 11%, respectively. Improvements were seen as early as 15 minutes after administration and lasted through 6 hours, based on measures on day 30.

New indications
Cortrophin Gel nabs expanded indications for certain chronic autoimmune disorders
ANI’s newly reintroduced Purified Cortrophin Gel (repository corticotropin intramuscular injection USP) has been granted new indications for treating certain chronic autoimmune disorders, including acute exacerbations of multiple sclerosis and rheumatoid arthritis, and excess urinary protein due to nephrotic syndrome. The treatment is for patients who are refractory or intolerant to corticosteroids.
The porcine-derived combination of adrenocorticotropic hormone and other peptides was first approved in 1954 and used for treating certain chronic autoimmune disorders. In addition to the new indications, it is also indicated for certain cases of collagen, dermatologic, ophthalmic, and respiratory diseases, as well as for edematous and allergic states.
ANI acquired the new drug application (NDA) for Cortrophin Gel after it bought Merck in 2016 and submitted a supplemental NDA to the FDA in June 2021.



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Tags: FDA, US

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