GeneOnline’s Pick: Top 10 New FDA-approved Drugs in 2022

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As part of the U.S. Food and Drug Administration (FDA), the Center for Drug Evaluation and Research (CDER) grants approval to new drugs or biologics in a variety of therapeutic areas every year. These products contain ingredients that have not been previously approved by the FDA and are important new therapies for patient populations in their respective areas.

By December 30, 2022, 37 drugs were approved by the FDA, covering a wide range of diseases including cancer, diabetes, blood disorders, autoimmune diseases, infections, and rare diseases. The following is a compilation of the top 10 FDA-approved drugs and biologics in various therapy areas selected by GeneOnline, with a review of key clinical data and mechanism of action (MOA), hoping to help readers to predict the development trend in 2023 by looking at novel drug approvals for 2022.

Related Article: GeneOnline’s Pick: Top 10 New FDA-approved Drugs in 2022

1. Idorsia’s Quviviq for Insomnia

On January 10, Swiss pharmaceutical company Idorsia announced that the FDA has approved Quviviq (daridorexant) for the treatment of adults with insomnia, characterized by difficulties with sleep onset and/or sleep maintenance. Following the success in the US, the drug also received marketing approval in Europe in April and in Switzerland in December. Quviviq is the first dual orexin receptor antagonist (DORA) approved as a treatment for insomnia in Europe and Switzerland, and it is available in Italy and Germany as of November. Besides, there is a phase 3 trial underway in Japan with positive results announced in October. 

Quviviq acts by binding two orexin receptors, OX1/OX2, in the brain to block the interaction of the wake-promoting neuropeptides orexins with their G protein-coupled receptors (GPCR). Such blocking action may turn down wakefulness and treat insomnia. Notable side effects of Quviviq include decreased awareness and alertness, as well as worsening of depressive symptoms and suicidal thoughts. Moreover, Quviviq is a federally controlled substance because it can be abused or lead to dependence.

Click here for more about the FDA approval of Quviviq.


2. Immunocore’s Kimmtrak for Unresectable or Metastatic Uveal Melanoma

Kimmtrak (Tebentafusp-tebn), a T cell receptor (TCR) therapeutic from the British biotechnology company Immunocore, won FDA approval on January 25 for the treatment of unresectable or metastatic uveal melanoma, a rare malignant tumor of the eye, It is the first TCR therapeutic as well as the first bispecific T cell engager for treating a solid tumor to receive regulatory approval from the FDA. 

As a bispecific protein, the T cell receptor end of Kimmtrak binds to gp100-HLA-A*02:01 on the surface of cancer cells, while the other end, anti-CD3 scFv, binds to and activates T cells with CD3, linking activated T cells to melanoma cells for cancer cell destruction. Results from the phase 3 clinical trial published in the New England Journal of Medicine showed that the 1-year overall survival for patients in the KIMMTRAK group was 73%, outperforming that of 59% in the control group which used other drugs including MSD’s Keytruda (pembrolizumab) and Bristol-Myers Squibb’s Yervoy (ipilimumab).

Click here for more about the FDA approval of Kimmtrak.


3. BMS’ Opdualag for Unresectable or Metastatic Melanoma

On March 18, Bristol Myers Squibb (BMS) announced that Opdualag, a new, first-in-class, fixed-dose combination of nivolumab (anti-PD-1) and relatlimab (a LAG-3 blocking antibody), administered as a single intravenous infusion, was approved by the FDA for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma.

The approval is based on the global phase 2/3 RELATIVITY-047 trial, which compared Opdualag to nivolumab alone. Given the similar safety profiles, the median progression-free survival (PFS) Opdualag was significantly higher than the nivolumab monotherapy, which is the established standard of care for existing patients (10.1 months vs. 4.6 months). Following the approval in the US, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave a positive recommendation for the European Commission in late July to approve the use of Opdualag in the European Union. Once approved, Opdualag will become the first LAG-3-blocking antibody combination in Europe.

Click here for more about the FDA approval of Opdualag.


4. Novartis’ Pluvicto for Prostate Cancer

On March 23, Novartis’ Pluvicto was granted FDA approval as a treatment of progressive, PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). Pluvicto is an intravenous radioligand therapy (RLT) that targets the Prostate Specific Membrane Antigen (PSMA). Pluvicto1L contains the PSMA inhibitor Vipivotide tetraxetan and the beta-emitting Lutetium (177Lu), which causes radiation damage to cancer cells when Vipivotide tetraxetan binds to them. 

In the pivotal Phase 3 VISION trial, Pluvicto significantly improved overall survival and reduced the risk of death by 38%, allowing the drug to win approval from the FDA. This approval also included Locametz (gallium Ga68 gozetotide), a companion diagnostic kit to Pluvicto which can identify tumor lesions expressing the PSMA biomarker and locate where in the body tumors may have spread, identifying patients eligible for targeted treatment with Pluvicto. In early December, Novartis announced that Pluvicto has achieved its primary endpoint in another Phase 3 study called PSMAfore in PSMA-positive mCRPC patients who have received androgen-receptor pathway inhibitor (ARPI) therapy, showing statistically significant and clinically meaningful radiographic progression-free survival (rFPS).

Click here for more about the FDA approval of Pluvicto.


5. Eli Lilly’s Mounjaro for Type 2 Diabetes

On May 13, Eli Lilly’s Mounjaro (tirzepatide), a novel glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist, received FDA approval as an adjunct to diet and exercise to improve blood sugar control in adults with type 2 diabetes. As the first and only FDA-approved GIP and GLP-1 receptor agonist, the drug represents a new class of type 2 diabetes medications in almost a decade.

Existing GLP-1 injection pens contain either liraglutide or semaglutide, both GLP-1 analogs that promote beta-cell growth, increase insulin secretion, and suppress appetite. In the 40-week Phase 3 SURPASS-2 trial, Mounjaro reduced glycohemoglobin (HbA1c) levels to a greater extent than semaglutide. In addition, Mounjaro showed greater efficacy in weight reduction in participants when compared with semaglutide. It is not known if Mounjaro can be used in people who have had inflammation of the pancreas (pancreatitis). Also, it is unknown whether the drug can be safely and effectively used by children under 18 years of age.

Click here for more about the FDA approval of Mounjaro.


6. Sanofi’s Xenpozyme for Acid Sphingomyelinase Deficiency 

On August 31, the FDA granted approval to Sanofi’s xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease that leads to premature death. ASMD is caused by the lack of an enzyme needed to break down a complex lipid called sphingomyelin, resulting in its accumulation in the liver, spleen, lung, and brain. The most severely affected patients have profound neurologic symptoms and rarely live beyond two to three years of age. Other patients may survive into adulthood but die prematurely due to respiratory failure.

As the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD, Xenpozyme is an enzyme replacement therapy that helps to metabolize sphingomyelin to achieve symptomatic relief. Clinical trial data have shown that Xenpozyme is effective in improving lung function and reducing liver and spleen volume in adults and children. However, because Xenpozyme is unable to cross the blood-brain barrier, it cannot reduce the accumulation of sphingomyelin in the brain.

Click here for more about the FDA approval of Xenpozyme.


7. Boehringer Ingelheim’s Spevigo for Generalized Pustular Psoriasis

On September 1, Boehringer Ingelheim announced the FDA approval of Spevigo (spesolimab-sbzo), a novel humanized anti–interleukin-36 (IL-36) receptor monoclonal antibody indicated for the treatment of generalized pustular psoriasis (GPP) flares in adults. 

GPP is a rare and potentially life-threatening neutrophilic skin disease that is characterized by an overproduction of IL-36 due to a loss of function mutation in the IL36RN and related genes, resulting in skin lesions. As the first approved treatment option for GPP flares in adults, Spevigo is able to block the activation of IL-36 receptor, a key part of a signaling pathway within the immune system shown to be involved in the cause of GPP, and hence provides symptomatic relief. In the 12-week Phase 2 pivotal Effisayil 1 trial, adult patients experiencing a GPP flare were treated with Spevigo or placebo. Approximately 54% of patients treated with Spevigo showed no visible pustules after one week compared to placebo (6%).

Click here for more about the FDA approval of Xenpozyme.


8. Eli Lilly’s Retevmo for Advanced or Metastatic Solid Tumors

Back in 2020, Eli Lilly’s Retevmo (selpercatinib) already secured FDA approval for the use in lung and thyroid cancers with RET gene mutations or fusions. On September 21, the FDA granted accelerated approval for Retevmo as the first and only RET Inhibitor for adults with locally advanced or metastatic RET fusion-positive solid tumors based on the positive results in the Phase 1/2 LIBRETTO-001 trial. The trial demonstrated clinically meaningful and durable responses across a variety of tumor types in patients with RET-driven cancers including pancreatic, colorectal, ovarian, and breast cancers, with an overall response rate (ORR) of 44% and a median duration of response (DOR) of 24.5 months. 

Apart from the above-mentioned accelerated approval, Retevmo also received a traditional approval from the FDA on the exact same day for its use in adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a RET gene fusion as detected by an FDA-approved test.

Click here for more about the FDA approval of Retevmo for RET fusion-positive solid tumors.


9. Taiho Oncology’s Lytgobi for Intrahepatic Cholangiocarcinoma

On September 30, 2022, the FDA granted accelerated approval to Lytgobi (futibatinib) for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (ICC) harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements. 

Only a minority (~15%) of patients with intrahepatic cholangiocarcinoma can receive surgical resection, with a median survival of 27-36 months, and about 10-16% of patients have FGFR2 recombinant fusion in their tumor cells. Lytgobi is an oral FGFR2 inhibitor that blocks signaling and stops cell growth. It has previously received breakthrough, orphan drug, and priority review designations from the FDA. The efficacy of the drug was evaluated in a Phase 2 TAS-120-101 trial involving 103 participants. The trial met its primary endpoint with an objective response rate of 42% as measured by an independent central review. The median duration of response (DOR) was 9.7 months, with 72% of responses lasting at least 6 months.

Click here for more about the FDA approval of Retevmo.


10. Provention Bio’s Tzield for Type 1 Diabetes

On November 17, Provention Bio’s Tzield (teplizumab-mzwv) received FDA approval as an injection to delay the onset of stage 3 type 1 diabetes in adults and pediatric patients 8 years and older who currently have stage 2 type 1 diabetes. 

Tzield is a humanized anti-CD3 antibody that does not bind to the Fc receptor, and modulates the immune system in a way that delays the progression of type 1 diabetes to stage 3. The drug’s safety and efficacy were evaluated in a Phase 2 trial with 76 patients with stage 2 type 1 diabetes, in which they randomly received Tzield or a placebo once daily for 14 days. Among the 44 patients treated with Tzield, 45% were later diagnosed with stage 3 type 1 diabetes compared to 72% of the 32 patients treated with placebo. The time taken for disease progression was 50 months in the Tzield group and 25 months in the placebo group. This represents a statistically significant delay of the onset of stage 3 type 1 diabetes.

Click here for more about the FDA approval of Tzield.

The following table is a summary of GeneOnline’s pick for top 10 New FDA-approved drugs in 2022:

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