Full Approval for Jemperli

Jemperli (dostarlimab-gxly – GSK) injection is a programmed cell death-1 (PD-1) inhibitor that received Accelerated Approval from the U.S. Food and Drug Administration (FDA) in April 2021. Its indication is for adults who have advanced endometrial cancer that previously has been treated with platinum-containing chemotherapy (chemo), that tests positive for deficient mismatch repair (dMMR) and that is not treatable with surgery or radiation. After the effectiveness and safety of Jemperli were confirmed by results from the continuing clinical trial, GARNET, the FDA upgraded Jemperli to full approval on Feb. 9, 2023. In the study, 45.4% of patients using Jemperli responded at least partially to therapy, with response times averaging 27.9 months. Administered by 30-minute intravenous (IV) infusions, the first four doses of Jemperli are given at 500mg once every three weeks. Three weeks after the fourth infusion, the dose and schedule shift to 1,000mg once every six weeks. Keytruda^® (pembrolizumab – Merck) is FDA-approved to treat dMMR-positive tumors that originate in multiple tissues, including the endometrium. Other PD-1-blocking antibodies that are available in the United States have indications for other types of dMMR-positive cancers, as well. Jemperli has another Accelerated Approval, which also will have to be verified, as second-or-later line therapy for dMMR-positive solid tumors that have no other treatment options. Here is its updated prescribing information.

Pediatric Indication Approved for Takhzyro

Takeda received FDA approval on Feb. 3, 2023, to expand the indication for Takhzyro^® (lanadelumab-flyo). A monoclonal antibody that blocks an enzyme involved in promoting pain and swelling, Takhzyro prevents symptoms associated with hereditary angioedema (HAE). In 2018, it was initially approved in the U.S. for patients aged 12 years old and older who have HAE, an orphan disease estimated to affect between 6,000 and 10,000 Americans. HAE is a genetic disorder caused by a deficiency of C1 inhibitor proteins. Patients who have it experience episodes of sudden, potentially life-threatening inflammation that cause swelling beneath the skin. The abdomen, arms and legs, face, hands and feet, throat, and urogenital tract all can be affected. Takhzyro is given by subcutaneous (SC) injections at doses of 150mg once every four weeks for children between the ages of two years old and six years old, 150mg once every two weeks for patients from six years old to 12 years old, and 300mg once every two weeks for those who are 12 years old and older. If HAE attacks are well controlled, dosing intervals may be lengthened to once every four weeks for some older children, teens, and adults. A healthcare provider or caretaker should administer Takhzyro for younger children, but adults and teens could be able to self-inject. Takeda’s C1 esterase inhibitor, Cinryze^®, also is approved for routine prophylaxis against HAE attacks for patients who are at least six years old. It is administered by intravenous (IV) infusion once every three or four days. Haegarda^® (C1 esterase inhibitor subcutaneous [human] – CSL Behring) is indicated for prevention, as well. It is injected SC – also once every three or four days and also for patients who are six years old and older. In December 2020, the FDA approved an oral medication, Orladeyo^™ (berotralstat – BioCryst Pharmaceuticals) capsules, to prevent HAE attacks for patients aged 12 years and older. It is taken once daily. Other drugs, including some C1 inhibitors, are FDA approved to treat acute attacks of HAE, but not to prevent them. For Takhzyro’s revised prescribing information, go here.

New Eylea Indication

Eylea^® (aflibercept – Regeneron) injection was FDA approved on Feb. 8, 2023, to treat retinopathy of prematurity (ROP) for babies who are born too early. Due to the underdevelopment of blood vessels in the retinas, as many as 1,500 premature babies need treatment for ROP, each year in the U.S. Detachment of the retinas and permanent loss of sight are possible results from untreated ROP. Previously, only a laser technique that seals leaking blood vessels was approved for treating it. Eylea blocks vascular endothelial growth factor (VEGF) to help prevent leaks from existing blood vessels and to lower the growth rate of new ones. Under sterile conditions, a healthcare professional injects it into the vitreous humor, the gel that fills the eyeballs. To treat ROP, the recommended dose is 0.4mg for each affected eye. Doses may be repeated once after 10 days if needed. In two phase III clinical trials, results and side effects from administering Eylea were comparable to those from using laser therapy. Eylea has prior approvals for treating adults who have neovascular age-related macular degeneration (nAMD), diabetic macular edema (DME), diabetic retinopathy (DR), and macular edema after retinal vein occlusion (RVO). Check here for its complete prescribing information.

Generic Available for Hetlioz

Teva recently introduced tasimelteon capsules to the U.S. market. It is the first generic for Vanda Pharmaceuticals’ Hetlioz^®, which was FDA approved in 2014 to treat adults who have the rare condition, non-24-hour sleep-wake disorder (non-24). Believed to affect as many as 70% of the roughly 1.3 million Americans who are at least 40 years old and who have complete or nearly complete vision loss, non-24 is a chronic circadian rhythm disorder involving the inability to synchronize the master body clock with the 24-hour day-night cycle. Patients who have it gradually fall asleep later at night, progressively delaying sleep onset and awakening times. Tasimelteon affects the body’s production of melatonin, helping to realign sleep cycles. The recommended dose is one capsule (20mg) each evening at the same time and about one hour before bedtime. Although Hetlioz has an additional indication for treating sleep disturbances associated with a developmental disorder known as Smith-Magenis Syndrome (SMS), the generic does not have approval for SMS. Tasimelteon capsules are not interchangeable with Hetlioz LQ oral solution, which does not have a generic. According to Vanda’s 2022 annual report, net sales for Hetlioz amounted to approximately $160 million for the calendar year.

Recalls
Tirosint-Sol
On Feb. 1, 2023, the FDA reported that IBSA Pharma Inc. recalled multiple lots of Tirosint-Sol at the consumer level. The recall included 27 lots of varying strengths and several different National Drug Codes (NDCs). The recall was initiated due to the potential that the lots may be less effective. If individuals with hypothyroidism (underactive thyroid) take Tirosint-Sol which is less effective, they may experience signs and symptoms of hypothyroidism, which can include, but are not limited to feeling tired, muscle weakness, slowed heart rate, sensitivity to cold, dry skin and weight gain. The FDA notice has more information.

Skippack COVID Tests

The FDA has reissued a recall for certain COVID test kits. On Feb. 8, 2023, it warned again that Skippack Medical Lab SARS-CoV-2 Antigen Rapid Test (Colloidal Gold) kits made by Universal Meditech were recalled last year to the patient level because they have not been approved or authorized for sale in the U.S. The results they provide may not be accurate — possibly missing cases of COVID-19 or falsely indicating active cases for patients who actually are not infected. Skippack tests were sold in the U.S. beginning in January 2022. More information about the recall is here. Patients who have recalled tests should not use them. For a complete list of COVID tests that have been cleared by the FDA, look here.