Your source for the latest industry trends and drug information news.
Volume 4 | Issue 24
December 15, 2022
Contributors:
Chief Authors: Rebecca Waite, PharmD, Bonnie Broczkowski, PharmD & Colleen Peehler, PharmD
Contributing Authors: Yi (Eve) Zheng, PharmD/MBA Candidate, Gabrielle Graca, PharmD/MBA Candidate
On November 30, 2022, the U.S. Food and Drug Administration (FDA) approved Rebyota® (fecal microbiota, live-jslm) to prevent the recurrence of Clostridioides difficile (C. diff) infection (CDI) in adults. C. diff is a harmful bacteria that can infect the colon, causing frequent episodes of watery diarrhea. Use of certain antibiotics increases the risk of developing a CDI because these antibiotics kill off the good bacteria in the colon that prevent an overgrowth of C. diff. Some C. diff infections can be serious and may be life-threatening. Once a person has experienced a CDI, they are at higher risk of reinfection. It has been estimated that 15% to 35% of CDI cases will result in recurrence, and people who have had a recurrence are at significantly higher risk for further C. diff infections. Rebyota® works by restoring the gut microbiome so good bacteria are available to prevent C. diff from reinfecting the colon.
Rebyota® is the first fecal microbiome therapy to be approved by the FDA. It is supplied as a single, pre-packaged rectal enema. The fecal matter used to manufacture the product is sourced from select donors and is carefully screened for infectious pathogens. In a clinical trial, 70.6% of subjects treated with Rebyota® had no further episodes of CDI diarrhea within 8 weeks of completing treatment, compared to 57.5% of those who received placebo. The treatment was generally well tolerated. The most common adverse effects were stomach pain, diarrhea and bloating. Rebyota® is only approved to prevent recurrence of CDI and is not approved for CDI treatment or for management of other gastrointestinal disorders.
On November 22, 2022, the FDA announced their investigation into the risk of severely diminished calcium levels in patients with advanced kidney disease who are taking the medication Prolia®. Prolia® (denosumab) is an injectable monoclonal antibody therapy that is administered once every six months in patients with osteoporosis. Reports from an ongoing clinical trial of Prolia® showed that patients with advanced kidney disease had an increased risk for low calcium levels which could lead to serious health outcomes, including hospitalization and death. Patients with advanced kidney disease requiring dialysis treatment have also shown a substantial risk for these outcomes while on Prolia®. Patients are recommended not to discontinue their Prolia® treatment without first consulting their health care provider to discuss the risks and benefits associated with continuing this treatment. Stopping Prolia® could worsen a patient’s bone condition, so it is important to consider alternative osteoporosis treatments. Patients with advanced kidney disease who continue on Prolia® therapy should supplement their dietary calcium and vitamin D intake, have their blood calcium levels tested regularly and watch for symptoms of low calcium levels. These symptoms include unusual numbness or tingling, irregular heart rhythm, voice box or lung spasms, painful muscle spasms or cramps, seizures and vomiting. Patients and health care professionals should report side effects of Prolia®, including low calcium levels, to the FDA MedWatch Adverse Event Reporting Program.
The FDA’s Accelerated Approval Program provides an avenue to encourage pharmaceutical manufacturers to develop urgently needed new medications and to make them available to patients as quickly as possible. The focus of this program is to address serious medical conditions for which there are limited or no treatments available. The drugs and vaccines considered for the Accelerated Approval Program include those developed to treat infectious diseases, cancer, and non-malignant blood and neurological disorders. Rather than requiring lengthy clinical trials for initial approval, accelerated approval is granted based on a surrogate endpoint, which is a marker that predicts clinical benefits. This can include physical signs, laboratory measurements, radiographic images or other measures. Although accelerated approval helps to shorten the time required before a medication is available to treat patients, the approval is only temporary. Pharmaceutical companies must conduct larger clinical trials following accelerated approval of the medication and provide the FDA with data from these trials to confirm the medication is effective. If these confirmatory trials are successful, the FDA will grant traditional approval for the drug or vaccine. If these confirmatory trials have unsatisfactory results and do not show clinical benefit, the FDA may remove the drug from the market.
On November 22, 2022, the pharmaceutical and biotechnology company GSK announced it would be withdrawing Blenrep (belantamab mafodotin-blmf) from the U.S. market. Blenrep received accelerated approval from the FDA in 2020 after presenting tumor shrinkage data in the treatment of multiple myeloma from two early trials. However, its confirmatory study, DREAMM-3, failed to meet its expected effectiveness endpoints and led to the withdrawal of the medication from the market. Several other treatments have had a similar outcome this year, including Ukoniq®, Marqibo® and Farydak®. Other medications, such as Tecentriq®, Zydelig® and Keytruda®, have remained on the market but have lost approval for some of their indications because confirmatory trials failed to show benefit for these conditions. Blenrep has two other studies in progress for use in combination with novel or standard medications to treat multiple myeloma, but GSK will need to submit a new biologics license application for FDA approval if the combinations are found to be effective.
New Indication: Brexafemme® (ibrexafungerp)
Oral tablet approved for the treatment of recurrent vulvovaginal yeast infection in adult and menstruating adolescent females. [11/30/2022 - SCYNEXIS]
Zioptan® (tafluprost)
Indication: Reduction of elevated pressure in the eye in adults
Dosage Form/Strength: 0.0015% Ophthalmic Solution
Average Wholesale Price (AWP): Generic = $262 | Brand = $276
