Omisirge Cell Therapy Receives FDA Approval
Gamida Cell received approval from the U.S. Food and Drug Administration (FDA) on April 17, 2023, for Omisirge® (omidubicel-onlv) suspension for infusion. Cell therapy is indicated for patients who are at least 12 years old and who need allogeneic (from a donor) hematopoietic stem cell (bone marrow) transplants to treat blood cancers. Gamida Cell uses banked umbilical cord blood that is altered using a form of vitamin B to increase and strengthen stem cells and natural killer (NK) cells in the transplant. Shipped frozen, specific protocols for thawing, diluting, and giving it will limit Omisirge’s use to hospitals and cancer centers that have staff trained in its administration and management of any side effects that may occur. Omisirge is given as one intravenous (IV) dose that contains a specific range of CD34+ cells after patients have been preconditioned with high doses of chemotherapy (chemo) that destroys cells in bone marrow (myeloablation). In a clinical trial, neutrophil (anti-infective white blood cells) counts for patients given Omisirge recovered in an average of 12 days versus 22 days for patients who received transplants derived from unaltered umbilical cord blood. About 39% of the patients who got Omisirge developed serious bacterial or fungal infections within 100 days of the transplant, as opposed to around 60% of those who got umbilical cord blood. Although donors are tested for underlying medical conditions, allogenic transplanted bone marrow may contain other types of cancer cells, infectious agents, or genetic abnormalities. Labeling for Omisirge has boxed warnings concerning the possibilities that it may cause life-threatening infusion reactions, graft versus host disease (GvHD), engraftment syndrome, or graft failure. An FDA-designated Orphan Drug, Omisirge was approved through the agency’s Breakthrough Therapy and Priority Review pathways. It will be excluded at launch on the Express Scripts National Preferred Formulary (NPF) until our formulary development process is complete. Here is its prescribing information.
New Dosage Form for Rizatriptan
An oral film version of rizatriptan, a treatment for acute migraines, was FDA-approved as a new drug on April 14, 2023. RizaFilm® VersaFilm® (IntelGenx) is indicated for patients who are 12 years old or older, who weigh 40kg (88 pounds) or more, and who have a diagnosis of migraines. The recommended dose is one film (10mg) allowed to dissolve on the tongue. If the headache is not relieved, doses for adults may be repeated at two-hour intervals with a limit of 30mg (three films) in a 24-hour period. Doses should not be repeated for pediatric patients. Like other serotonin (5-HT) 1B/1D receptor agonists (triptans), RizaFilm works by reducing blood vessel dilation and inflammation in the brain. Several other oral, nasal, injected, and transdermal triptans are FDA-approved, many with generic equivalents. Triptans do not prevent migraines and they do not treat cluster headaches. The launch date and cost for RizaFilm are not yet available. Express Scripts currently is investigating the data around RizaFilm for a possible utilization management strategy. It also will be excluded at launch on the Express Scripts National Preferred Formulary (NPF) until our formulary development process is complete. Look here for its full prescribing information.
Extended Indication for Qulipta
The indication for AbbVie’s oral calcitonin gene-related peptide (CGRP) receptor antagonist, Qulipta® (atogepant) tablets was extended by the FDA on April 17, 2023. First approved in September 2021 to prevent episodic migraine headaches for adults, it now is approved for preventing chronic migraines for adults, as well. Episodic migraines occur between four days and 14 days per month; chronic migraines affect patients on 15 or more days a month. The recommended dose for chronic migraine prevention is 60mg once a day. By the fifth week in the 12-week-long clinical trial for the new indication, patients taking Qulipta averaged about two fewer headaches per month as compared to those taking placebo tablets. All study participants were allowed to use pain relievers for treating migraines. The most commonly experienced adverse effects were nausea, constipation, and fatigue. Ubrelvy® (ubrogepant – AbbVie) and Nurtec® ODT (rimegepant – Pfizer) are oral CGRP inhibitors approved to treat migraines once the headaches have started. In June 2021, Nurtec ODT received an additional indication to prevent migraine attacks. A nasal spray, Zavzpret™ (zavegepant) was approved in March 2023, to treat, but not to prevent migraines. Three monoclonal antibody CGRP inhibitors — Aimovig® (erenumab-aooe – Amgen), Ajovy® (fremanezumab-vfrm – Teva Pharmaceuticals USA) and Emgality® (galcanezumab-gnlm – Eli Lilly) were FDA approved in 2018 to prevent migraines. All three are self-injected subcutaneously (SC), typically once a month. Approved in 2020, another monoclonal CGRP inhibitor, Vyepti® (eptinezumab-jjmr – Lundbeck), requires IV administration once every three months to prevent migraines. Updated prescribing information for Qulipta is here.
New Indication and Full Approval for Polivy
Polivy® (polatuzumab vedotin-piiq), a novel antibody-drug conjugate made by Genentech, was FDA approved on April 18, 2023, as initial treatment for a second subset of patients who have diffuse large B-cell lymphoma (DLBCL). It now can be used along with R-CHP, a chemo regimen that includes rituximab, cyclophosphamide, doxorubicin, and prednisone; for treatment-naïve patients whose DLBCL is not otherwise specified (NOS), or high-grade with an International Prognostic Index (IPI) score of two or more. On a scale from one to five (worst prospect), IPI estimates the patient’s chances of surviving five years or longer. In June 2019, Polivy originally received the FDA’s Accelerated Approval as a third- or later-line treatment for DLBCL along with rituximab and bendamustine. That indication now has been upgraded to full approval, based on confirmatory results from the POLARIX clinical trial. In the study, about 77% of patients who got the Polivy/R-CHP regimen had no disease progression at an average of 28.2 months compared to around 70% of patients receiving R-CHOP, chemo that includes vincristine along with the R-CHP drugs. For both indications, Polivy is administered at 1.8mg/kg as IV infusions on the first days of six 21-day cycles. Using it may be associated with numerous possible side effects including bone marrow suppression, infusion reactions, liver damage, and peripheral neuropathy. Serious infections may be more likely for patients, as well. Look here for Polivy’s complete prescribing information.
FDA Updates COVID-19 Vaccination Recommendations
On April 18, 2023, the FDA made significant revisions to streamline its recommendations for COVID-19 vaccinations. The changes were supported by the Center for Disease Control (CDC) Advisory Committee on Immunization Practices (ACIP) on the following day. The emergency use authorizations (EUAs) for the original, monovalent vaccines from Moderna and Pfizer/BioNTech have been canceled. Those vaccines no longer will be recommended in the U.S., although monovalent Novavax and Janssen vaccines continue to be available for individuals who choose not to receive an mRNA-based vaccination. Additionally, adults who previously have not been vaccinated for COVID-19 will need only one dose of the current, bivalent vaccines. Children between the ages of six months and five years old still will need two (Moderna) or three (Pfizer/BioNTech) immunizations for their initial vaccinations, however. Noting that infections generally were not as severe for patients who got COVID after having boosters last fall, the FDA recommends one booster with a bivalent product for all individuals who are six years old and older. They also advise that older adults and individuals who are immunocompromised may be eligible for a second booster, but that boosters should be separated by at least four months for individuals who are 65 years old or older and by at least two months for those who have immunocompromising conditions. In June, an FDA committee will decide which strains of COVID-19 should be included in the next versions of the vaccines/boosters, much like the composition of influenza (flu) vaccines is re-evaluated each year. For current EUAs, Fact Sheets, and other detailed information on Moderna’s vaccine, look here; for Pfizer/BioNTech’s, here.