Pulse Newsletter | Volume 8 - Issue 10

FDA Approves First in Class Drug to Treat Metastatic Pancreatic Cancer

On August 26, 2026, the U.S. Food and Drug Administration (FDA) approved Rasonque^™ (daraxonrasib) for the treatment of adult patients with metastatic (spread to other parts of the body) pancreatic cancer who have previously received at least one prior treatment or for patients who are not candidates for multiagent chemotherapy. Rasonque^™ is the first medication for pancreatic cancer that inhibits multiple forms of RAS, a protein produced in the pancreas that facilitates tumor growth. The safety and efficacy of Rasonque^™ was studied in an open-label, multicenter clinical trial including 500 patients with metastatic pancreatic cancer. Patients received either Rasonque^™ 300mg once daily or standard of care chemotherapy. Patients treated with Rasonque^™ had a statistically significant improvement in their overall survival (13.2 months vs. 6.7 months), progression-free survival (7.2 months vs. 3.6 months) and tumor response rate (30% vs. 11%) compared to standard of care chemotherapy.

The recommended dose of Rasonque^™ is 300mg taken by mouth once a day with or without food. Warnings and precautions include risk of severe skin damage that may include symptoms of itching, rash and dry skin. To reduce the risk of skin reactions, patients are advised to use preventative treatments including applying topical steroid creams (to face and chest) and moisturizing creams, along with limiting time spent in the sun and applying sunscreen (SPF 30 or higher). Preventative treatment with oral antibiotics may also be considered. Other precautions include risk of ulcers in the mouth, perforation of the stomach, nausea, diarrhea, fatigue, the worsening of lung disease and the risk of harm to an unborn baby. The list price of Rasonque^™ is $39,800 per month.

FDA Provides an Update on Estradiol Patch Availability

On September 3, 2026, the FDA provided an update on the ongoing supply issues impacting generic estradiol patches. Estradiol patches are FDA-approved for the treatment of vasomotor symptoms (including hot flashes and night sweats) and genitourinary symptoms due to menopause. During menopause, the body stops producing certain hormones, including estrogen, which can lead to various vasomotor symptoms and bone loss, increasing the risk for fracture. Women can experience the various symptoms of menopause for up to 10 years, with the average age of menopause occurring at 52 years. Demand for estradiol patches increased following a drug safety update announced by the FDA in November 2025 advising pharmaceutical manufacturers to make a change to their product labeling based on a review of current clinical evidence. The FDA requested that the boxed warning (the strongest safety warning for prescription medications) for probable risk of breast cancer, heart disease and dementia be removed from the package labeling for several menopausal hormone therapy (MHT) products, including estrogen and progesterone prescription products (single and combination products). The resultant increase in prescribing of MHT has led to a shortage of estradiol patches.

Drug manufacturers are working on increasing drug supply for estradiol patches to keep up with the increase in demand, and patients are advised to check with their dispensing pharmacy to determine current availability. Patients who are still experiencing ongoing supply issues or shortages of their prescribed estradiol patches should contact their doctor or pharmacist to determine if an alternative estradiol product is available.

FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy

On September 11, 2026, the FDA approved Isembyld^™ for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years and older who are currently on standard therapy. SMA is a rare genetic disease that affects motor neurons and causes progressive muscle weakness and muscle wasting. Isembyld^™ is administered by a healthcare professional as an intravenous injection once every 4 weeks. Dose is based on weight, with a recommended dose of approximately 4.5 mg per pound of body weight. Prior to administration, Isembyld^™ must be diluted in 0.9% sodium chloride. Warnings and precautions of Isembyld^™ include serious risk of bone fracture. The most common side effects seen with Isembyld^™ therapy are cough, vomiting, infections (including upper respiratory infections and other viral infections), headache, inflammation of the lining of the stomach and intestines, sore throat and hypersensitivity reaction. Isembyld^™ should not be used during pregnancy due to risk of harm to an unborn baby.

The safety and efficacy of Isembyld^™ was established in a 52-week randomized, double-blind, placebo-controlled multicenter clinical trial with 188 patients diagnosed with SMA who could not walk on their own. The primary endpoint of the trial was change in patient’s motor function compared to baseline. Patients in the Isembyld^™ group were more likely to achieve a clinically meaningful improvement in their motor ability compared to the placebo group (34.2% vs. 13.5%). The annual cost for Isembyld^™ is expected to be approximately $310,000, although price may vary as dosing is based on patient weight.

New FDA Approvals

New Drug: Etcamah^® (camizestrant)

Oral tablets approved for the treatment of adult patients with a specific type of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer. [9/4/2026 – AstraZeneca Pharmaceuticals LP.]

New Generics Entering the Marketplace

Bexlutry^™ (lutetium Lu 177 dotatate)

Indication: For the treatment of adult patients with a specific type of advanced neuroendocrine tumor (a rare type of cancer that primarily affects cells in the pancreas and digestive tract). BEXLUTRY^™ is the first FDA-approved radioligand equivalent. Radioligand therapies use very small amounts of radioactive material to destroy cancer cells.  

Dosage Form/Strength: 370MBq/mL solution for injection

Average Wholesale Price (AWP): Generic = $76,2000/vial | Brand = $80,450/vial