According to a recent article in Medscape, the FDA has issued accelerated approval for Bristol-Myers Squibb’s Pomalyst (pomalidomide).
Pomalyst was approved as the first new oral treatment for AIDS-related Kaposi sarcoma (KS) in over twenty years. In addition, the drug has its own protocol which is unlike the chemotherapy drugs used in the past to treat KS.
Pomalyst has similar properties to thalidomide and is currently being marketed to treat multiple myeloma.
The drug specifically targets KS that are resistant to HAART which is Highly Active Antiretroviral Therapy that is a combination of three or more drugs to treat HIV infection.
Pomalyst also treats KS occurring in HIV-negative patients. It has therefore shown positive results in patients whether they are positive or negative for HIV.
The Basis for FDA Approval
Conditional FDA approval for Pomalyst was based on a seventy-one percent overall response stemming from a clinical trial (Phase 1/2) that involved twenty-eight patients.
Of the twenty-eight participants, eighteen were HIV-positive with ten patients were HIV negative. The primary endpoint of the trial was the overall response rate. Fifty-seven percent of patients had partial responses while fourteen percent of the patients had a complete response.
Fifty percent of patients who registered either a partial or full response continued those responses for over twelve months.
Trial participants were administered 5mg of Pomalyst one time each day for twenty-one of a twenty-eight-day cycle. The dosing continued until or unless patients experienced severe toxicity or the disease progressed.
Out of twenty-eight patients, only three had to leave the trial due to adverse reactions.
About Adverse Reactions
Common reactions to Pomalyst may include one or more of the following symptoms: maculopapular rash with raised skin lesions, nausea, constipation/diarrhea, fatigue, difficulty breathing (dyspnea), upper respiratory tract infection, hypothyroidism, chills, muscle spasms and peripheral edema (swelling of hands or legs).
Pomalyst issues a boxed warning in its prescribing information because it can cause birth defects and/or embryonic fetal death.
Other possible symptoms may be a pulmonary embolism, myocardial infarction, stroke, and deep vein thrombosis. Patients were given eighty-one mg of aspirin every day to avoid blood clots. There were no new safety issues reported.
A spokesperson for Bristol-Myers noted that for over twenty years, patients with this rare disease had very few options for disease management.
FDA Contingent Approval
The company will conduct a confirmatory trial to meet the FDA’s requirements for verification and further proof of the drug’s clinical benefit.
According to the FDA, HIV patients with AIDS-related KS may continue with HAART in accordance with their doctor’s instructions.
Pomalyst is only available through restricted distribution.
