belzutifan

Belzutifan (Welireg, Merck & Co., Inc.) was approved by the Food and Drug Administration on December 14, 2023.

Belzutifan belongs to a group of drugs known as hypoxia-inducible factor inhibitors.

Scotland has become the first European country to approve the use of MSD’s WELIREG (belzutifan), an HIF-2?± inhibitor, for eligible adults with von Hippel-Lindau (VHL) disease within the NHS.

Merck’s Belzutifan in Phase II for Von Hippel-Lindau Syndrome: Prospects for Approval. Merck is currently conducting clinical trials for Belzutifan, a drug aimed at treating Von Hippel-Lindau Syndrome, and is in Phase II of development.

The cobas EGFR Mutation Test is a PCR test that uses real-time technology to detect specific mutations in the EGFR gene in patients with non-small cell lung cancer (NSCLC).

At present, the sole stem cell therapy that undergoes regular evaluation and endorsement by the U.S. Food and Drug Administration (FDA) is hematopoietic (or blood) stem cell transplantation. This treatment is employed to address the medical needs of patients suffering from blood-related cancers and immune system disorders.

The Food and Drug Administration has granted approval to pembrolizumab (Keytruda, Merck) for use as neoadjuvant treatment in combination with platinum-containing chemotherapy, and as post-surgical adjuvant treatment for resectable non-small cell lung cancer (tumors measuring ?4 cm or with positive lymph nodes). This approval was given on October 16, 2023.

The FDA has given approval for a number of serum tumor markers, such as Carcinoembryonic antigen (CEA), alpha-fetoprotein (AFP), prostate specific antigen (PSA), CA 125 (residual epithelial ovarian cancer), and soluble Interleukin-2 (IL-2) receptor.

Elrexfio, a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, has been granted accelerated approval by the Food and Drug Administration on August 14, 2023. This approval is for the treatment of adults with relapsed or refractory multiple myeloma who have undergone at least four prior lines of therapy. The manufacturer of Elrexfio is Pfizer, Inc.

Due to the high occurrence of clear cell renal cell carcinoma linked to von Hippel-Lindau (VHL) disease, individuals affected by this condition typically have a life expectancy of 49 years. Nevertheless, vigilant monitoring and prompt treatment can extend life expectancy.

The disease has a prevalence of 1 in 36,000 live births and has a high penetrance rate of 90% by the age of 65. One of the most common symptoms of VHL is the development of cerebellar lesions, which typically occur around the age of 30. A diagnosis of VHL is confirmed when there is a positive family history of the disease and the presence of at least one VHL-related tumor.

Renal cell carcinoma, the most prevalent form of kidney cancer, is developed by approximately 40% of individuals with VHL disease. This is also the primary cause of disease-related mortality in those with VHL. Renal cell carcinoma can manifest as a solitary tumor in one kidney or as multiple tumors in both kidneys.

Von Hippel-Lindau disease is incurable, but the focus of treatment is on early detection and removal of tumors to prevent health complications. Surgery may be recommended to remove tumors, and some tumors may also be treated with radiation therapy.

Symptoms include blood in the urine, a lump or growth on the side or lower back, fever, weight loss, back pain, decreased appetite, and fatigue.

Welireg (belzutifan) has been granted orphan drug status in the UK, limiting its availability to a specific patient population rather than being widely accessible on the market.

Drug Company Indication
Darzalex Janssen Biotech Multiple Myeloma
Soliris Alexion Pharmaceuticals Paroxysmal Nocturnal Hemoglobinuria, Hemolytic Uremic Syndrome, Myasthenia Gravis
Venclexta Abbvie Chronic Lymphocytic Leukemia, Acute Myeloid Leukemia
Jakafi Incyte Corporation Myelofibrosis

You can find Belzutifan in 40 mg tablets sold under the brand name Welireg. The suggested daily dosage is 120 mg until either disease progression or if there are any adverse effects that are not tolerable.

The FDA has recently designated three new drugs as orphan drugs. These include oral fludarabine phosphate for chronic lymphocytic leukemia, clobazam for Lennox-Gastaut syndrome, and oral beclomethasone dipropionate for pediatric Crohn’s disease.

Ultra-orphan drugs refer to medications that are specifically designed to treat extremely rare diseases that have a chronic and debilitating impact on individuals or pose a life-threatening risk. Due to the limited number of patients affected by these diseases, pharmaceutical companies face challenges in recovering the costs associated with researching and developing these drugs. As a result, these medications tend to be expensive when considering the cost per patient.

An orphan drug refers to a specific type of medication designed to address rare medical conditions. The production of an orphan drug would not be financially viable without government support, primarily because the number of patients affected by these conditions is relatively small.

By December 31, 2022, the Office of Orphan Products Development (OOPD) had issued 6340 orphan drug designations, covering 1079 different diseases. Over the period from 1983 to 2022, a total of 1122 orphan-designated products received approvals, including approvals for new molecules, indications, and formulations.

WELIREG is approved for the treatment of adult patients with von Hippel-Lindau (VHL) disease who need therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET) and do not require immediate surgery.

In addition, belzutifan demonstrated a higher overall response rate of 22% compared to 3.5% with everolimus. Furthermore, 13 patients achieved a complete response with belzutifan, while none experienced this outcome with everolimus. Additionally, patients receiving belzutifan were less likely to discontinue treatment due to side effects.

Some of the most frequent side effects of WELIREG are headaches, dizziness, elevated blood sugar levels, and nausea.

Welireg, also known as belzutifan, belongs to the miscellaneous antineoplastics drug class and is frequently prescribed for Von Hippel-Lindau Syndrome. The price of a 90-tablet supply of Welireg oral tablets at a pharmacy can vary, but typically costs approximately $29,755. Patients may be able to save money on their medication through the use of coupons or patient assistance programs.

Belzutifan, an inhibitor of hypoxia-inducible factor 2? (HIF-2?), is utilized for treating cancers associated with von Hippel-Lindau (VHL) disease. The discovery of the HIF-2? protein’s significance in the development of specific cancers was made by UT Southwestern Medical Center researchers in the 1990s.

The FDA approved belzutifan (WELIREG, Merck) on August 13, 2021. It is the first-in-class hypoxia-inducible factor (HIF) inhibitor for adult patients with von Hippel-Lindau (VHL) disease who need treatment for associated renal cell carcinoma (RCC) and central nervous system (CNS) tumors.

The adverse reactions that occurred in more than 25% of patients who received WELIREG included decreased hemoglobin, anemia, fatigue, increased creatinine, headache, dizziness, increased glucose, and nausea.

Before starting Welireg, it is important to confirm whether the patient is pregnant or not, as the drug has the potential to cause harm to the embryo and fetus. This warning is highlighted in the Black Box Warning section of the Welireg drug label.

Belzutifan, marketed as Welireg, is an oral anti-cancer drug prescribed for the management of renal cell carcinoma associated with von Hippel-Lindau disease. It functions as an inhibitor of hypoxia-inducible factor-2 alpha (HIF-2?±).

VHL syndrome has a prevalence of one in 36,000 individuals, making it a relatively rare condition. As VHL syndrome is a genetic disorder, there is a possibility that your family members may also carry the mutation. In fact, around 80% of cases are inherited from a parent who already has VHL syndrome. Fortunately, advancements in tumor detection techniques have significantly enhanced the care provided for individuals with VHL syndrome.

Surgery is a frequently utilized treatment for various problems associated with VHL, which may involve minimally invasive techniques or laser surgery in certain cases. However, the success of surgery for VHL disorders greatly relies on the expertise of a specialist who has extensive experience in performing the specific procedure.

VHL can only be diagnosed through genetic testing, which will reveal a genetic mutation in the VHL gene for almost all individuals with the condition. However, there are currently no standardized recommendations for screening individuals for VHL.