A Quick Review of the Major FDA Drug Approvals in November 2023

Approvals from the United States Food and Drug Administration include drugs for various rare diseases. Furthermore, drug treatment approval for desmoid tumours, breast cancers, metastatic ROS1-positive non-small cell lung cancer (NSCLC), and metastatic colorectal cancer (mCRC) are also on the list of approvals this month.

Eli Lilly's Jaypirca Received a Green Flag for Treating Chronic Lymphocytic Leukemia

On Dec 1st, 2023, Eli Lilly announced the approval of its drug Jaypirca by the U.S. Food and Drug Administration (FDA). This drug is used in the treatment of a blood cancer known as chronic lymphocytic leukaemia (CLL). In this medical condition, the bone marrow makes too many of certain white blood cells. Nearly 87% of people suffering from chronic lymphocytic leukaemia (CLL) survive for 5 or more years following diagnosis. CLL accounts for approximately one-quarter of new leukaemia cases, and while it is rare for treatment to cure CLL, it can be successfully managed.

The FDA also granted an accelerated approval for Jaypirca  earlier this year for treating mantle cell lymphoma (MCL), a rare type of blood cancer that starts in white blood cells and spreads to other parts of the body.

Desmoid Tumours Get the First Ever Drug Treatment with Nirogacestat

The first-ever drug treatment for desmoid tumours was approved by the Food and Drug Administration in late November 2023. The drug named Nirogacestat (OGSIVEO), developed by SpringWorks Therapeutics, Inc., was approved for adult patients with progressing desmoid tumours who require systemic treatment. The recommended dose for Nirogacestat is 150 mg, which is to be administered orally twice daily. Each 150 mg dose consists of three 50 mg tablets.

Desmoid tumours, also known as aggressive fibromatosis, are noncancerous growths occurring in the connective tissue. The most common sites of encounter of desmoid tumours include abdomen, arms, and legs. Some desmoid tumours progress gradually and hence do not require immediate treatments. However, others show quick progression and are treated with surgery, radiation therapy, chemotherapy, or other drugs.

Efficacy for Nirogacestat was evaluated in DeFi (NCT03785964), an international, multicentre, randomized (1:1), double-blind, placebo-controlled trial in 142 patients with progressing desmoid tumours not amenable to surgery. Patients were eligible if the desmoid tumour had progressed within 12 months of screening. Patients were randomized to receive 150 mg Nirogacestat or placebo orally twice daily until disease progression or unacceptable toxicity.

The most common adverse reactions observed among patients were diarrhoea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia , upper respiratory tract infection , and dyspnoea.

Desmoid tumours are considered rare, however, about 900 to 1,500 people in the United States are expected to be diagnosed with a desmoid tumour each year. This type of tumour commonly affects people between the ages of 15 and 60 years, but it can occur at any age. Desmoid tumours are more common in women than in men. Due to them being considered noncancerous, they can show aggressive traits, acting more like cancers and growing into nearby structures and organs.

AstraZeneca's Truqap to be Used as a New Breast Cancer Treatment Alternative

The U.S. Food and Drug Administration has approved AstraZeneca's Truqap in combination with an older drug, providing another treatment option for patients with the most common type of breast cancer . Clinical trials have shown that the approved drug combination can help these patients live without disease progression for an additional 7.3 months. Truqap belongs to a class of drugs known as AKT inhibitors and works by targeting the AKT1 gene mutation, which is responsible for the growth and proliferation of tumours. Some of the common side effects of the drug include diarrhea, decreased haemoglobin, nausea, and fatigue, among others.

This FDA approval permits the usage of Capivasertib drug in combination with the British drugmaker's older cancer treatment Faslodex. With this approval, the Truqap combination enters a competitive breast cancer treatment market,  including AstraZeneca-Daiichi Sankyo's Enhertu and Gilead's (GILD.O) Trodelvy.

 
According to the American Cancer Society, nearly, 297,790 new cases of invasive breast cancer are expected to be diagnosed in women this year. Furthermore, around 55,720 new cases of ductal carcinoma in situ (DCIS) are expected to be diagnosed, and breast cancer is expected to claim almost 43,700 lives. The FDA has approved a drug combination that is effective in treating patients with breast cancer of the HR-positive/HER2-negative subtype. The drug can also be used in patients whose tumours have alterations in one of three genes, namely PIK3CA, AKT1, or PTEN.

Bristol-Myers Squibb Company's Repotrectinib Approved for Treating Metastatic ROS1-positive Non-small Cell Lung Cancer

On November 15th, 2023, the Food and Drug Administration approved repotrectinib (Augtyro) byBristol-Myers Squibb Company for treating locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). This is the first FDA approval that includes patients with ROS1-positive NSCLC who have recently received a ROS1 tyrosine kinase inhibitor (TKI), in addition to patients who are TKI-naïve.

According to the American Cancer Society, the estimates for lung cancer  in the US for 2023 include about 238,340 new cases of lung cancer (117,550 in men and 120,790 in women). Furthermore, lung cancer cases are expected to claim nearly 127,070 lives (67,160 in men and 59,910 in women). 

New Hope in Treating Metastatic Colorectal Cancer with Takeda Pharmaceutical's Fruquintinib

Takeda Pharmaceuticals, Inc., on November 8th, 2023, announced that the US FDA approved their Fruquintinib (Fruzaqla), for adult patients with metastatic colorectal cancer  (mCRC). This drug is administered to patients who have received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. The recommended Fruquintinib dose is 5 mg orally once daily, with or without food, for the first 21 days of each 28-day cycle until disease progression or unacceptable toxicity.

Colorectal cancer begins in the colon or rectum, however, metastatic colorectal cancer  spreads to regional or distant sites, such as other organs or lymph nodes. It is the third most common cancer accounting for approximately 10% of all cancer cases worldwide and is the second accounting for leading cause of cancer-related deaths worldwide. It is commonly diagnosed at advanced stages when treatment options are often limited.

Efficacy was evaluated in FRESCO-2 (NCT04322539) and FRESCO (NCT02314819). FRESCO-2 (NCT04322539), an international, multicentre, randomised, double-blind, placebo-controlled study, evaluated 691 patients with mCRC who had disease development during or after prior fluoropyrimidine-, oxaliplatin-, irinotecan-based chemotherapy,  FRESCO, a multicentre, placebo-controlled study conducted in China, evaluated 416 patients with mCRC who had disease progression during or after prior fluoropyrimidine-, oxaliplatin and irinotecan-based chemotherapy.

The most common side effects experienced (in ≥20% of patients) by the patients include hypertension, palmar-plantar erythrodysesthesia, proteinuria, dysphonia, abdominal pain, diarrhoea, and asthenia.

These FDA approvals are expected to give hope to healthcare professionals in the treatment of rare and incurable medical conditions such as Chronic Lymphocytic Leukemia (CLL). Furthermore, these approvals are expected to fetch some major opportunities for the key players in the market to expand their portfolios by manufacturing these treatment drugs.