Cinematic October 2019 FDA Approvals
My 14-year-old son and I are movie buffs. Each week, we check the theater’s schedule to see what is being released. We even check the ‘flashback’ cinema offerings to see what great movie of the past is returning to the big screen for a quick showing. Occasionally, a plot will catch my eye as being a truly innovative storyline. The recent trend, though, has been to ‘re-make’ what already exists, with perhaps a new take or a new angle on the theatrics or storyline. We enjoy both. We love new, innovative storylines, but we also love comparing the new re-makes to the traditional movies that we know so well.
When I take off my ‘dad hat’ and put on my ‘work hat’, my attention is drawn to things like new drug approvals and industry trends. As different as the worlds of Hollywood and medicine are, I find myself using the same evaluative lens. Some approvals are truly novel, others are an interesting ‘re-make’, and a few will open an entirely new avenue for treatment. And, as with great movies, the final place in history may not be realized for some time. Which will eventually be those ‘classic products’ worthy of bringing back to the ‘big screen’ (or to the treatment stage, if you will) in 20 years? As we look at a sampling of the October 2019 Food and Drug Administration (FDA) approvals, then, I encourage you to pick up this theatrical lens with me:
October 2019 FDA approvals included the approval of Galderma Laboratories’ Aklief® (pronounced ack-LEEF), a 0.005% trifarotene cream indicated for the topical treatment of acne vulgaris in patients 9 years of age and older. Two identical 12-week, randomized, multicenter, parallel group, double-blind, vehicle-controlled clinical trials of 2,420 patients showed a success rate of 29.4% (versus 19.5% for placebo / vehicle cream) and 42.3% (versus 25.7% for placebo / vehicle cream) respectively.
The cream is applied each evening to the affected areas on clean and dry skin. The concurrent use of a moisturizer is recommended. Trifarotene selectively targets the retinoic acid receptor gamma. As with other topical retinoid therapies, patients should be advised to minimize exposure to sunlight and to use sunscreen when exposure cannot be avoided. The most common adverse reactions included application site irritation and sunburn. Alkief® is expected to be available in the United States in November 2019.
Eli Lilly & Company received approval for Reyvow™ (lasmiditan), which introduces a new class of oral medication indicated for the acute treatment of migraine with or without aura in adults. Lasmiditan is a serotonin (5-HT) 1F receptor agonist, so the usual advisement applies of discontinuing the medication should symptoms of serotonin syndrome emerge. The efficacy was demonstrated in two randomized, double-blind, placebo-controlled trials. Patients reporting being ‘pain free’ or ‘free of the most bothersome symptom’ represented a variance from placebo between 10.4% and 11.6% for a 100mg dosage and between 11.1% and 17.8% for a 200mg dosage.
The recommended dosage is 50mg – 200mg by mouth as needed, with no more than one dose administered in a 24-hour period. Patients should be advised to refrain from driving or operating machinery for at least 8 hours after dosing. Additionally, patients should be aware of the potential for ‘medication overuse headache’ which results from the overuse of acute migraine drugs, typically defined as use for more than 10 days each month.
Vertex Pharmaceuticals announced the fast track approval of Trikafta® (elexacaftor/ivacaftor/tezacaftor), a triple-combination therapy consisting of three different modulators. Trikafta® has been approved for patients with cystic fibrosis aged 12 and older who have at least one copy of the F508del mutation, which is estimated to represent 90% of the cystic fibrosis population. Currently available therapies that target the defective protein are not viable options for many patients.
Efficacy analysis in two clinical trials evaluated the percent predicted forced expiratory volume in one second (ppFEV1). In the first trial, Trikafta® increased mean ppFEV1 13.8% from baseline compared to placebo. In the second trial, Trikafta® increased mean ppFEV1 10% from baseline compared to tezacaftor/ivacaftor. Trikafta® received an ‘orphan drug’ status, an incentive to encourage the development of drugs for rare diseases.
Trikafta® comes with warnings related to elevated liver function tests and the risk of premature development of cataracts. The product is dosed twice daily with fat-containing food.
So, there it is: a sampling of the ‘newest releases’ for October. What do you think? Any interesting ‘re-makes’? Any novel pieces? Anything that stands out to shape that way that we practice medicine? Surely something resonated in a familiar way using this lens? If not, stay tuned. Hollywood and the FDA are both bustling places full of surprises.
Kevin T. Hope, RPh is the Director of Continuing Education with the PharmCon team in Myrtle Beach, SC. He is a graduate of the Medical University of South Carolina and has served as adjunct faculty for the South Carolina College of Pharmacy, having coordinated the college’s authorized user program in nuclear pharmacy. In addition to academic experience with both pharmacy students and pharmacy technicians, Kevin brings an array of perspectives from many facets of the profession, including retail, hospital, and nuclear pharmacy experiences.