Pulse Newsletter | Volume 7 - Issue 21

FDA Approves First Oral Targeted BTK Inhibitor Treatment for Patients with Chronic Hives

On September 30, 2025, the U.S. Food and Drug Administration (FDA) approved Rhapsido® (remibrutinib) for the treatment of adult patients with chronic spontaneous urticaria (CSU), also known as chronic hives, who have already been treated with antihistamines and remain symptomatic. Hives are considered chronic if they appear at least two times per week and last for at least 6 weeks. Symptoms of chronic hives include itching, inflammation and raised red welts on the skin surface. The exact cause of CSU is not known, but it may be due to an autoimmune process. Of the estimated 1.7 million Americans diagnosed with CSU, more than half continue to experience symptoms despite antihistamine therapy. Currently other therapies indicated for the treatment of chronic hives, in addition to antihistamines, include the injectable monoclonal antibody drugs, Dupixent® (dupilumab) and Xolair® (omalizumab).

Rhapsido® is the first oral Bruton’s tyrosine kinase (BTK) inhibitor available for the treatment of chronic hives. The safety and efficacy of Rhapsido® was evaluated based on two 52-week, double-blind, placebo-controlled clinical trials, REMIX-1, and REMIX-2 with 912 adult participants. Patients treated with Rhapsido® experienced improvement in their disease as seen by a reduction in itching and hives. Side effects seen with Rhapsido® therapy include the common cold (including inflammation of the nose and throat, nasal congestion, sore throat, coughing), bleeding, headache, nausea and stomach pain. A warning associated with Rhapsido® includes risk of bleeding, and patients should be monitored for this adverse effect. It is also recommended that patients taking Rhapsido® should avoid receiving live vaccines. Certain other classes of drugs, including CYP3A4 inhibitors and CYP3A4 inducers, should be avoided if using Rhapsido®. Patients taking P-gp substrates, such as digoxin, should be monitored closely as small changes in drug concentration may lead to more serious side effects. Rhapsido® is expected to be priced at around $4,500 for a 30-day supply and will be available in Q4 of 2025.

FDA Announces New ANDA Prioritization Pilot to Support Domestic Generic Drug Manufacturing and Testing

On October 3, 2025, the FDA announced a new pilot program for the priority review of generic drugs seeking an abbreviated new drug approval (ANDA) manufactured inside the United States. The program allows pharmaceutical manufacturers the ability to qualify for priority review if they manufacture their drug and conduct any required generic bioequivalence testing in the U.S., along with using only domestic supplied active pharmaceutical ingredients (APIs). According to the FDA, the U.S. currently manufactures less than half of all pharmaceutical drugs that are distributed nationally. In addition, according to the FDA, U.S.-based manufacturers represent only a minority (less than 10% as of 2025) of all manufacturers of APIs used in FDA-approved medications. APIs are the component of a drug responsible for its therapeutic effect, while the other inactive ingredients may be used for things like absorption, stability, binding, or drug delivery in the body.

Approval by the FDA is required prior to a drug being marketed in the U.S. There are multiple types of drug applications that can be submitted to the FDA for approval. A new drug application (NDA) must be submitted by pharmaceutical companies when seeking FDA approval for a medication with a new active ingredient, compared to an abbreviated new drug application (ANDA), which can be submitted for a generic drug. A generic drug contains the same active ingredient as the original brand name drug and can enter the market once the patent has expired for the original brand name drug. An NDA application is more extensive than an ANDA and requires submission of detailed clinical trial data demonstrating the drug’s safety and efficacy profile, along with an assessment of risks versus clinical benefits of therapy. An ANDA typically requires only demonstration of bioequivalence to the original drug since the safety and efficacy have been established through the original drug’s NDA. Generic drugs are generally more cost effective compared to brand name drugs, therefore, expanding domestic supply and access to safe and effective generic drugs is a priority for the federal government and the FDA. This new program, along with several other new programs recently announced — including the FDA’s PreCheck program — are designed to incentivize and boost drug manufacturing in the United States.

FDA Approves New Treatment for Acromegaly

On September 25, 2025, the FDA approved Palsonify™ (paltusotine) tablets for the treatment of adult patients with acromegaly who did not respond to, or cannot have, surgery. Acromegaly is a rare condition in adults when the pituitary gland produces an excessive amount of growth hormone, typically due to a non-cancerous pituitary tumor. Patients experience abnormal growth in bones, organs, and other tissues which can lead to enlarged hands and feet, facial changes, thickened skin, and joint pain. If left untreated, patients with acromegaly can develop serious and sometimes life-threatening complications.

The safety and efficacy of Palsonify™ was evaluated in two randomized, double-blind, placebo-controlled Phase 3 drug trials of adults with acromegaly. The primary endpoint was control of acromegaly, as measured by insulin-like growth factor 1 (IGF-1) levels. IGF-1 is a hormone involved in growth and metabolism. The first study enrolled 111 adult patients with acromegaly. At week 24, 56% of patients treated with Palsonify™ had control of their disease, as indicated by IGF-1 levels, compared to 5% of patients treated with placebo. The second study enrolled 58 adult patients with acromegaly. At week 36, 83% of patients treated with Palsonify™ had control of their disease via IGF-1 levels compared to 4% of patients treated with placebo.  

Palsonify™ is supplied as an oral tablet and is taken once daily. Warnings and precautions of Palsonify™ include risk of gall stones, cardiovascular and thyroid abnormalities, fat malabsorption, vitamin B-12 deficiency and fluctuations in blood sugar. The most common adverse effects seen with therapy with Palsonify™ include gastrointestinal side effects, such as nausea, diarrhea, stomach pain, inflammation of the stomach and decreased appetite. Other side effects include high blood sugar, heart palpitations and reduced heart rate (bradycardia). Drug interactions with Palsonify™ include CYP3A4 inducers, proton pump inhibitors and cyclosporine. The anticipated monthly cost of Palsonify™ is approximately $24,000 per information available from the manufacturer and it is expected to be available in Q4 2025.

New FDA Approvals

New Drug: Clotic® (clotrimazole)

Ear solution approved for the treatment of adult patients with a fungal infection of the ear canal due to Aspergillus species or Candida species. [9/26/2025 – Laboratorios Salvat, S.A.]

New Generics Entering the Marketplace

Ravicti® (glycerol phenylbutyrate)

Indication: For management of patients with urea cycle disorders (UCDs) not controlled by dietary restrictions

Dosage Form/Strength: Oral liquid

Average Wholesale Price (AWP): Generic = $6,387/bottle | Brand = $7,097/bottle