Your source for the latest industry trends and drug information news.
Volume 5 | Issue 10
June 15, 2023
Contributors:
Chief Authors: Rebecca Waite, PharmD, Bonnie Broczkowski, PharmD & Colleen Peehler, PharmD
Contributing Authors: Yi (Eve) Zheng, PharmD/MBA Candidate, Gabrielle Graca, PharmD/MBA Candidate
On May 19, 2023, the U.S. Food and Drug Administration (FDA) announced the approval of Vyjuvek™ for the treatment of wounds associated with dystrophic epidermolysis bullosa (DEB) in adult and pediatric patients 6 months and older. DEB is a rare genetic disorder that results from the mutation of a gene involved in the production of COL7A1, a protein essential in strengthening and stabilizing layers of the skin and nails. When inadequate amounts of COL7A1 are present, there may be separation between the layers of the skin, causing blisters and wounds. Symptoms often appear at birth and can affect individuals at various levels of severity. Patients with dominant DEB (DDEB) may experience mild blistering of the knees, elbows, hands and feet while those with recessive DEB (RDEB) may experience much more blistering. This blistering can become debilitating and extremely painful, leading to disfigurement, vision loss, other medical complications and potentially death.
Vyjuvek™ is a gene therapy that is applied to a patient’s wounds once weekly by a health care professional. It uses a genetically modified herpes-simplex virus (HSV) to bring working copies of COL7A1 genes to blisters and wounds. COL7A1 proteins are produced at the site, holding the layers of the skin together. Vyjuvek™ was studied in a randomized, double-blinded, placebo-controlled clinical trial of 31 individuals with DEB. Patient ages ranged from 1 to 44 years. All but one participant presented with the RDEB type. Efficacy was measured by comparing the proportion of complete wound closure between patients treated with Vyjuvek™ versus the placebo group. After 24 weeks, 65% of participants treated with Vyjuvek™ had complete wound closure, compared to only 26% of those participants receiving placebo. The most reported adverse reactions were redness, rash, itching, chills, cough and runny nose. Vyjuvek™ received Orphan Drug, Fast Track, Priority Review and Regenerative Medicine Advanced Therapy designations. It also received an FDA Rare Pediatric Disease Priority Review Voucher.
On May 18, 2023, Novis PR LLC announced their voluntary recall of 1 lot of G-Supress DX Pediatric Drops. G-Suppress DX Pediatric Drops are oral drops that contain a combination of medications used to treat cold symptoms like cough and congestion. Some cartons of Lot D20911 of the pediatric drops have been found to contain an oral anesthetic/analgesic rather than the intended product. This anesthetic/analgesic product contains 60% ethyl alcohol and 5% benzocaine. Ingestion of these ingredients could increase the risk of alcohol toxicity in infants and young children. Small amounts of ethyl alcohol could lead to low blood sugar, hypothermia, coma or even death in this population. Affected cartons display an expiration date of 10/25. Novis PR LLC is notifying distributors and customers of this recall and arranging for the return of recalled products. At this time, there have been no reported adverse events or injuries related to this mispackaging. Consumers may contact their physician if they have experienced any problems related to this recall. Additionally, adverse reactions may be reported to the FDA’s MedWatch Adverse Event Reporting Program.
The Centers for Medicare and Medicaid Services (CMS) announced on June 1, 2023, that they will broaden their coverage of Alzheimer’s slowing drugs for Medicare patients upon receiving full FDA approval. Two drugs, Aduhelm® (aducanumab-avwa) and Leqembi™ (lecanemab-irmb), have received accelerated FDA approval based on evidence of a reduction in the amyloid plaques in the brain associated with Alzheimer’s Disease. Medicare coverage of these medications is currently restricted to beneficiaries enrolled in clinical trials. In their announcement, CMS stated that anyone participating in a Medicare Part B plan who has been diagnosed with mild cognitive impairment or early dementia caused by Alzheimer’s disease will be covered for these treatments once they receive traditional approval from the FDA. On June 9, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee reviewed the results of the most recent clinical trial of Leqembi™ and voted unanimously to recommend full approval for the drug. The FDA’s decision is expected by early July. No date has been projected for approval consideration of Aduhelm®. Once traditional approval is granted and Medicare B coverage is broadened, patients treated with an approved drug will be required to participate in a HIPAA compliant, CMS-facilitated registry which will help patients and their clinicians determine if these treatments are appropriate for them. The registry will also collect evidence about how the newly approved drugs work in application by tracking data on safety and effectiveness. The information gathered will help further the understanding of how these treatments can potentially benefit patients.
New Drug: Inpefa™ (sotagliflozin)
Oral tablet approved to reduce the risk of cardiac events and death in adult patients with heart failure or with type 2 diabetes mellitus, chronic kidney disease or other cardiovascular risk factors. [05/26/2023 – LEXICON PHARM INC]
New Drug: Miebo™ (perfluorohexyloctane)
Eye drops approved for the treatment of signs and symptoms of dry eye disease. [05/18/2023 – BAUSCH AND LOMB INC]
Celontin® (methsuximide)
Indication: Absence, or petit mal, seizures that do not respond to other drugs
Dosage Form/Strength: 300mg oral capsule
Average Wholesale Price (AWP): Generic = $291 | Brand = $324
