This quarterly review on new drug applications contain data for applications approved for the first time during the second quarter of 2019 which includes new molecular entities (NMEs) and new biologics. A total of 30 applications were approved by FDA during these three months (April-June).
Following are the percentages of various dosage form in this quarter - 23.3% tablets, 10% capsules, 46.7% injectables, 6.6% non-injectable solutions, and 13.4% miscellaneous. Miscellaneous dosage forms include chewable bars, lotions, and nasal sprays. There are two tentatively approved products under 505(b)(2) application.
Colesevalam (Welchol), used to treat elevated LDL level, is available as tablets (675 mg per tablet) or powder for suspension (3.75 g per pouch). Daiichi Sankyo has introduced a chewable bar (3.75 g/ bar). The chewable bar contains ~ 80 calories and it is available in chocolate, strawberry and caramel flavors.
Tafamidis is the first FDA approved treatment for cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTRCM). Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare and fatal condition that is caused by destabilization of a transport protein called transthyretin, which is composed of four identical sub units (a tetramer). When unstable transthyretin tetramers dissociate, they result in misfolded proteins that aggregate into amyloid fibrils and deposit in the heart, causing the heart muscle to become stiff, eventually resulting in heart failure. There are two sub-types of ATTRCM; hereditary which is caused by mutation in the transthyretin gene and wild type which is associated with aging. Prior to the approval, the only available option was symptom management and in rare cases, heart or liver transplant.
Ruzurgi is the second FDA-approved treatment for LEMS (Lambert-Eaton Myasthenic Syndrome) and the first approved treatment for pediatric LEMS patients. LEMS is a rare autoimmune disorder in which the body’s own immune system attacks the neuromuscular junction (the connection between nerves and muscles) and disrupts the ability of nerve cells to send signals to muscle cells. Ruzurgi is an oral potassium channel inhibitor designed to prolong signals released from nerves to allow greater stimulation of muscle.
In June 2019, FDA granted accelerated approval to Polivy in combination with Bendamustine plus Rituxan for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma. The FDA's Accelerated Approval Program allows earlier approval of a medicine that fills an unmet medical need for a serious condition based on a surrogate end point. According to section 507(e)(9) of the FD&C Act, the term ‘surrogate endpoint’ means a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure, that is not itself a direct measurement of clinical benefit. Drug companies are still required to conduct studies to confirm the clinical benefit. These studies are known as Phase 4 confirmatory trials. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug.
Duobrii lotion is a combination of halobetasol propionate and tazarotene and it is used to treat psoriasis. Halobetasol is a corticosteroid and tazarotene is a member of acetylenic class of retinoid. The lotion contains carbomer copolymer type B, carbomer homopolymer type A, diethyl sebacate, edetate disodium dihydrate, light mineral oil, methylparaben, propylparaben, purified water, sodium hydroxide, sorbitan monooleate and sorbitol solution, 70%. This lotion may cause fetal harm if administered to pregnant women.