Cuvrior Approved to Treat Wilson’s Disease

A new form of trientine, Cuvrior™ (trientine tetrahydrochloride – Orphalan) was approved on April 28, 2022, by the U.S. Food and Drug Administration (FDA). It is indicated for treating adults who can no longer take penicillamine to treat Wilson’s disease and who have been “de-coppered” (treated so that their blood levels of copper are within normal ranges). A rare inherited disorder, Wilson’s disease causes the accumulation of copper in the body. Fewer than 3,000 patients have been diagnosed with Wilson’s disease in the United States, but numbers may be higher because its symptoms, such as bruising, fatigue and swelling, are similar to several other conditions. Untreated Wilson’s disease can severely damage the liver and central nervous system (CNS), potentially causing death. First-line drug choices for treating it are penicillamine (available as the brands Cuprimine® capsules and Depen® tablets, as well as generics) or trientine hydrochloride (Clovique™ capsules, Syprine® capsules and generics). They are chelating agents that help the body eliminate copper. Once blood levels of copper are within normal limits, maintenance therapy can continue with penicillamine, trientine hydrochloride or Galzin® (zinc acetate) capsules, which blocks the absorption of copper from food. Up to one-third of patients cannot continue using penicillamine, however, due to possibly serious side effects that can include blood abnormalities, joint pain and nausea. Cuvrior is a new option for maintenance. It is recommended for twice daily dosing in two equal parts that total between 300mg per day and 3,000mg per day depending on the patient’s copper levels. It will be available as 300mg tablets in the first part of 2023. Pricing is not yet available. Trientine hydrochloride products are not directly interchangeable with Cuvrior.

FDA Approves New Drug to Treat Helicobacter pylori

Phathom Pharmaceuticals received FDA approval on May 3, 2022, for its first drug, vonoprazan. Only for use in combination with antimicrobial drugs, it is indicated for treating adults who have Helicobacter pylori (H. pylori) infections. The first drug in its class, vonoprazan is a potassium-competitive acid blocker (PCAB) that reduces stomach acid in a different way than other available drugs do. For commercial sale, it will be blister packaged along with the antibiotic amoxicillin as Voquenza™ Dual Pak™ (vonoprazan tablets, 20mg/amoxicillin capsules, 500mg) and along with both amoxicillin and another type of antibiotic, clarithromycin, as Voquenza™ Triple Pak™ (vonoprazan tablets 20mg/amoxicillin capsules/500mg and clarithromycin tablets 500mg). Each package contains a complete 14-day treatment regimen. With the triple therapy performing slightly better, the overall effectiveness of both vonoprazan regimens was comparable in clinical studies to results from the three-drug combination of lansoprazole, amoxicillin and clarithromycin, which is widely used to treat H. pylori. For patients who had clarithromycin-resistant H. pylori, both vonoprazan combinations were more effective than the lansoprazole combination. (65.8% for the triple therapy, 69.6% for dual and 31.9% for the lansoprazole combination). Side effects, which included diarrhea, headaches, taste disturbances and vaginal infections, affected fewer than 5% of patients in any of the study groups. Launch is planned for the third quarter of 2022. Cost information has not yet been released. Both Voquenza versions were given Priority Review as qualified infectious disease products (QIDP), an FDA program that promotes the development of antibacterial and antifungal drug products that treat serious or life-threatening infections.

Rinvoq Receives New FDA Approval

AbbVie won FDA approval on April 29, 2022, for Rinvoq® (upadacitinib) extended-release tablets to treat active cases of ankylosing spondylitis (AS) for adult patients. A selective inhibitor of Janus-associated kinase (JAK), it blocks signal transmissions to interrupt inflammation. To treat AS, its recommended dose is 15mg once a day. Rinvoq’s previous FDA-approved indications are as late-line therapy for specific patients after treatment with at least one tumor necrosis factor (TNF) blocker, such as Humira® (adalimumab), has failed to manage atopic dermatitis (AD), psoriatic arthritis (PsA), rheumatoid arthritis (RA) or ulcerative colitis (UC). Rinvoq should not be taken at the same time as a biologic disease-modifying anti-rheumatic drugs (DMARDs), immunosuppressants or other JAK inhibitors are being used. A boxed warning is required by the FDA for all JAK inhibitors that are used to treat inflammatory conditions. It cautions that patients taking them may have a greater chance of dying, developing certain cancers, suffering cardiovascular (CV) events, having blood clots and acquiring severe infections. Patients also should be screened for tuberculosis (TB) before starting treatment with a JAK inhibitor and periodically while therapy continues.

Extended Indication for Qelbree

On April 29, 2022, the FDA approved Qelbree® (viloxazine – Supernus Pharmaceuticals) extended-release capsules to treat adults who have attention deficit hyperactivity disorder (ADHD). It is a non-stimulant drug originally approved in April 2021, for treating children between the ages of six years and 18 years who have ADHD. For patients who are at least 18 years old, the recommended starting dose of Qelbree is 200mg once a day. If more control is needed, daily doses can be increased by 200mg on a weekly basis to an upper limit of 600mg per day. Labeling for Qelbree has a boxed warning that children and adolescents who take it may have a higher risk for suicidal ideas and/or acts, so they should be watched for unusual behavioral changes during treatment.

Full Approval and Expansion for Enhertu’s Breast Cancer Indication

The breast cancer indication for Enhertu® (fam-trastuzumab deruxtecan-nxki) for injection, which was granted in December 2019 under the FDA’s Accelerated Approval process, was fully approved on May 4, 2022. It is for treating adults who have metastatic breast cancer that is positive for human epidermal growth factor receptor 2 (HER2+) and that is inoperable or that metastasizes further after at least two previous treatments with anti-HER2 drugs. Additionally, the use of Enhertu was widened to include adults whose unresectable or metastatic HER2+ breast cancer has returned within six months of anti-HER2-based treatment either in the metastatic setting or before (neoadjuvant) or after (adjuvant) initial treatment, which usually is surgery Enhertu is a conjugate that links an antibody specific to HER2 with a topoisomerase inhibitor that kills cancer cells. In the U.S., it is marketed jointly by Daiichi Sankyo and AstraZeneca Pharmaceuticals. For treating breast cancer, the recommended dose is 5.4mg/kg infused intravenously (IV) once every three weeks until the cancer stops responding to the drug or the side effects become unbearable for the patient. Enhertu also has approval for treating adults who have locally advanced or metastatic HER2+ gastric or gastroesophageal junction (GEJ) adenocarcinoma that previously was treated with trastuzumab. Because it may cause potentially fatal lung conditions and it also may damage a developing baby, Enhertu has boxed warnings and a patient Medication Guide.

MedWatch Update – Counterfeit COVID-19 Tests

In a safety communication sent on April 29, 2022, the FDA reminded consumers to be sure that non-prescription COVID-19 tests are genuine before buying them. Some COVID-19 test kits that are being sold in the U.S. have not been evaluated as correct and have not been approved, even though some may be in packages that are similar to tests that are authorized by the FDA. The accuracy of the counterfeit tests cannot be assured, so their results cannot be trusted. Consumers should check for clues, such as printing or spelling errors and missing information, such as lot numbers and barcodes, on the test boxes. More information is in the FDA’s notice and in a website that includes data on all COVID-19 tests that are FDA approved.

Johnson & Johnson COVID-19 Vaccine

Rare cases of thrombosis with thrombocytopenia syndrome (TTS), which is blood clots along with low blood platelet levels, have occurred after vaccination with the COVID-19 vaccine made by the Janssen division of Johnson & Johnson (J&J). Most cases appeared within two weeks after the shot was given. Although the risk of TTS is extremely small at around three cases per one million vaccine recipients, the FDA determined that use of the vaccine should be restricted. On May 5, 2022, it released a decision that the J&J vaccination should be administered only to adults who would not be vaccinated for COVID-19 because they cannot take or they decline to have other available vaccines. Currently, the J&J vaccine has an emergency use authorization (EUA) not yet a full FDA approval. For additional details, please see the FDA notice, the Fact Sheet for Prescribers or the Fact Sheet for Recipients.

Recalls – Glenmark Generics

After an FDA inspection of a Glenmark Pharmaceuticals manufacturing facility questioned the ability of their microbiology lab procedures to assure product sterility, Glenmark recalled several generics to the consumer level on April 25, 2022. Contamination in drugs could result in potentially life-threatening infections. The recalls include:

• arformoterol inhalation solution, a long-acting beta agonist indicated for maintenance treatment of chronic obstructive pulmonary diseases (COPD)
• chlorzoxazone tablets, a drug that relaxes muscles
• naproxen tablets, a non-steroidal anti-inflammatory drug (NSAID) for managing pain
• zonisamide tablets, which treats epilepsy