Innovation drives progress. When it comes to innovation in the development of new drugs and therapeutic biological products in the pharmaceutical industry, the United States Food and Drug Administration’s (FDA’s) Center for Drug Evaluation and Research (CDER) is a key supporter of the pharmaceutical industry.  

CDER relies on its understanding of science and regulatory procedures to support and provide advice to pharmaceutical companies in testing and manufacturing, to then bring these new therapies to the market to aid patients and their health.

The FDA protects public health by ensuring the efficacy and safety of human and veterinary drugs, including biological products and medical devices. It is critical for the FDA to continue to develop new advances in therapies to aid our communities in order to achieve better lives through better health.

Below we will be listing some of the new drug therapy advances and approvals that the FDA had in the month of September.

Lytgobi 

• Date of Approval: 9/30/2022

Lytgobi (futibatinib) is an irreversible tyrosine kinase inhibitor of FGFR1, 2, 3, and 4 which was discovered by Taiho Oncology, Inc’s parent company, Taiho Pharmaceutical. The FDA has approved Lytgobi tablets for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 gene fusions or other rearrangements.

This indication was approved under accelerated approval based on the response and duration of the response. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials. 

Common adverse effects include musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, and abdominal pain, among others.

 

Relyvrio 

• Date of Approval: 9/29/2022

Relyvrio (sodium phenylbutyrate and taurursodiol) previously known as AMX0035 in the U.S., is a neuroprotective therapy for the treatment of amyotrophic lateral sclerosis (ALS). The approval of Relyvrio provides another treatment option for patients battling with ALS, a threatening disease that has no cure at the moment. 

The FDA granted the approval of Relyvrio to Amylyx Pharmaceuticals Inc. following data from the CENTAUR Phase 2 Clinical Trials. 

Common adverse effects noted include diarrhea, abdominal pain, nausea, and upper respiratory tract infection.

 

Omlonti

• Date of Approval: 9/22/2022

Omidenepag isopropyl is the active ingredient in Omlonti and it is a relatively selective prostaglandin E2 receptor agonist. Its mechanism is due to an increase in aqueous humor drainage through the conventional and uveoscleral outflow pathways.

Omlonti is the only drug with this particular pharmaceutical mechanism pathway. 

Common adverse reactions include conjunctival/ocular hyperemia, photophobia, blurred vision, dry eye, pain, headache, irritation, and visual impairment.

 

Elucirem

• Date of Approval: 9/21/2022

Elucirem (gadopiclenol) is a macrocyclic gadolinium-based contrast agent (GBCA) for use with MRI to detect and visualize lesions with abnormal vascularity in the CNS and the body. It is indicated for use in adults and children aged 2 years and older. 

The approval was primarily based on data from two Phase III studies which demonstrated that Elucirem leads to non-inferior results in brain and body MRI at half the gadolinium dose of Gadobutrol of existing non-specific GBCAs (gadolinium-based contrast agents). This addresses practitioners’ concerns about gadolinium exposure. 

Common adverse effects observed included injection site pain, headache, nausea, dizziness, and localized swelling.

 

Terlivaz

• Date of Approval: 9/14/2022

Terlivaz (terlipressin) is a vasopressin receptor agonist indicated to improve kidney function in adults with hepatorenal syndrome (HRS) with a rapid reduction in kidney function. Terlivaz is the first and only FDA-approved product indicated to improve kidney function in adults with HRS with a rapid reduction in kidney function, an acute and life-threatening condition requiring hospitalization.

Its FDA approval was announced by Mallinckrodt plc. This approval was based on results from the Phase 3 CONFIRM trial which assessed the safety and efficacy of terlipressin in patients with HRS type 1. 

Common adverse effects include serious or fatal respiratory failure, ischemic events, abdominal pain, nausea, and diarrhea, among others.

 

Rolvedon

• Date of Approval: 9/9/2022

Rolvedon (eflapegrastim-xnst) is a leukocyte growth factor used to reduce the incidence of infection as manifested by febrile neutropenia, in adults with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia

Rolvedon was supported by data from two Phase 3, randomized, open-label, non-inferiority clinical trials, ADVANCE, and RECOVER, which evaluated safety and efficacy. In both studies, Rolvedon demonstrated the pre-specified hypothesis of non-inferiority in the mean duration of severe neutropenia and a similar safety profile to pegfilgrastim. 

Serious adverse effects observed include kidney injury, allergic reactions, nausea, headache, rash, and decreased red blood cell count, among others.

Sotyktu

• Date of Approval: 9/9/2022

Sotyktu (deucravacitinib) is a tyrosine kinase 2 (TYK2) inhibitor indicated for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. Bristol Myers Squibb’s Sotyktu is first-in-class, and it is the only approved TYK2 inhibitor worldwide.

Pivotal Phase 3 POETYK PSO clinical trials demonstrated superior efficacy of once-daily Sotyktu over placebo and twice-daily Otezla® (apremilast) in improving skin clearance. 

Common adverse effects include upper respiratory infections, blood creatine phosphokinase increased, herpes simplex, mouth ulcers, folliculitis, and acne.

 

Daxxify

• Date of Approval: 9/7/2022

Daxxify (DaxibotulinumtoxinA-lanm) was approved for the temporary improvement of moderate to severe frown lines (glabellar lines) in adults. It is the first and only neuromodulator stabilized with Peptide Exchange Technology.

The U.S. approval of Daxxify was based on the SAKURA Phase 3 clinical trial program (SAKURA 1,2,3), where 74% of subjects achieved a greater than two-grade improvement in glabellar lines at week 4. 

Common adverse effects observed were headache, eyelid ptosis, and facial paresis, including facial asymmetry.

 

Spevigo

• Date of Approval: 9/1/022

Spevigo (spesolimab-sbzo) is the first approved treatment option for generalized pustular psoriasis (GPP) flares in adults. Spevigo is a selective antibody that blocks the activation of the interleukin-36 receptor, a key part of a signaling pathway within the immune system shown to be involved in the cause of GPP.

FDA approval follows the 12-week Effisayil 1 clinical trial, 53 patients experiencing a GPP flare were treated with Spevigo or a placebo. After one week, patients treated with Spevigo showed no visible pustules (54%) compared to placebo (6%). 

Common adverse effects were asthenia and fatigue, nausea and vomiting, headache, pruritus and prurigo, infusion site hematoma and bruising, and urinary tract infection.

 

For more up-to-date information on these approvals, visit fda.gov.