As of now, the FDA has approved some major new drugs in 2024, including Eli Lilly's Kisunla for Alzheimer's disease, Madrigal Pharmaceuticals' Rezdiffra for metabolic dysfunction-related fatty liver disease, and BMS's first new schizophrenia drug in 35 years, KarXT, among others...

In December, several new drugs were approved. Below is a summary of the drugs approved by the FDA in December.

Zepbound

Developing Company: Eli Lilly

Indication: Moderate to severe obstructive sleep apnea in obese adults

Eli Lilly's Zepbound (tirzepatide) is already a major player in the weight loss field. Recently, the FDA announced that Zepbound is now approved for treating moderate to severe obstructive sleep apnea in obese adults, and it should be used in combination with diet and exercise.

The FDA's approval was based on two randomized, double-blind, placebo-controlled studies—one group used Positive Airway Pressure (PAP) treatment, while the other group did not. A total of 469 participants without type 2 diabetes were enrolled. After 52 weeks of treatment with Zepbound (10 or 15 mg) or placebo once a week, patients on the drug experienced statistically and clinically significant reductions in apnea or hypopnea events, along with weight loss. The FDA noted this could help improve symptoms of apnea.

Alyftrek

Developing Company: Vertex Pharmaceuticals

Indication: Cystic fibrosis in adults and children aged 6 years and above with at least one F508del mutation or another CFTR gene mutation responsive to treatment

Vertex Pharmaceuticals announced that the FDA approved its new cystic fibrosis drug Alyftrek, a modification of Trikafta. Trikafta generated $8.9 billion in sales in 2023.

Alyftrek is a triple combination therapy consisting of Vanzacaftor, Tezacaftor, and Deutivacaftor. Vanzacaftor and Tezacaftor aim to increase the amount of CFTR protein on the cell surface by promoting its processing and transport, while Deutivacaftor improves the probability of channel opening, facilitating better salt and water flow across the cell membrane. The FDA approval was based on positive results from three Phase III studies.

Alhemo

Developing Company: Novo Nordisk

Indication: Hemophilia A or B in adults and children aged 12 years and above

Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist for subcutaneous injection. Recently, Novo Nordisk's Alhemo was approved for preventing bleeding episodes or reducing bleeding frequency in adults and children aged 12 and above with hemophilia A or B and inhibitors to clotting factors.

This approval was supported by a key trial showing that Alhemo reduced the frequency of spontaneous and traumatic bleeding by 86% compared to the control group.

Ensacove

Developing Company: Xcovery

Indication: ALK-positive locally advanced or metastatic non-small cell lung cancer

Xcovery Holdings' Ensacove (ensartinib) has been approved by the FDA for the treatment of ALK-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) as a first-line therapy.

ALK is a gene primarily involved in development that is typically turned off before birth. In some cases, it is reactivated and fuses with neighboring genes, resulting in various cancers, including lymphoma and NSCLC. Ensacove is an oral tyrosine kinase inhibitor that targets ALK-mediated signaling, blocking the abnormal growth and proliferation of cancer cells.

Steqeyma

Developing Company: Celltrion

Indication: Plaque psoriasis, psoriatic arthritis in adults and children, Crohn's disease, and ulcerative colitis in adults

Following the FDA approval of Celltrion's biosimilar Steqeyma, Johnson & Johnson’s Stelara (ustekinumab) will face more intense competition. Steqeyma is expected to launch in the U.S. in February 2025, in subcutaneous and intravenous forms.

The approval of Steqeyma was based on Phase III study results for plaque psoriasis, which showed that the biosimilar had no clinically meaningful differences in efficacy or safety compared to Stelara.

Nemluvio

Developing Company: Galderma

Indication: Moderate to severe atopic dermatitis

The FDA has approved Galderma's subcutaneous injection, Nemluvio (nemolizumab-ilto), for patients aged 12 and older with moderate to severe atopic dermatitis. It is recommended to be used in combination with topical corticosteroids and/or calcineurin inhibitors.

Nemluvio is a humanized IgG2 monoclonal antibody targeting the IL-31 receptor, inhibiting downstream signaling and reducing inflammation, fibrosis, and epidermal dysregulation. Its approval was supported by the Phase III ARCADIA clinical trial, which showed significant improvements in skin clearance and reduction in itching and sleep disturbances compared to a placebo.

Vtama

Developing Company: Organon

Indication: Atopic dermatitis

The FDA approved Organon's topical cream Vtama (tapinarof) for the treatment of atopic dermatitis. Vtama is suitable for both adult patients and children as young as 2 years old.

Vtama is an aryl hydrocarbon receptor agonist. It was initially approved in May 2022 for plaque psoriasis. The approval is supported by clinical trial results showing Vtama’s superior efficacy in helping patients achieve clear or almost clear skin compared to placebo, with durable effects and good tolerability during the 48-week follow-up.

Crenessity

Developing Company: Neurocrine

Indication: Congenital adrenal hyperplasia

Neurocrine’s Crenessity (crinecerfont) has been approved as the first new treatment for congenital adrenal hyperplasia (CAH) in 70 years. CAH is a rare genetic disorder characterized by a deficiency in essential adrenal steroid hormones.

Crenessity selectively blocks CRF1 receptors, helping regulate ACTH and other adrenal androgens. Its approval is supported by data from the Phase III CAHtalyst study, which showed significant reductions in dehydroepiandrosterone sulfate (DHEAS) levels in patients.

Unloxcyt

Developing Company: Checkpoint Therapeutics

Indication: Metastatic or locally advanced cutaneous squamous cell carcinoma

The FDA has approved Checkpoint Therapeutics' PD-L1 inhibitor Unloxcyt for the treatment of metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC).

Unloxcyt is the first and only PD-L1 blocker approved for this indication. It acts by blocking the interaction between PD-L1 and its receptors, PD-1 and B7.1, thus enhancing the anti-tumor immune response. The approval was supported by data showing objective response rates of 47.4% in metastatic patients and 54.8% in locally advanced patients.

Imfinzi

Developing Company: AstraZeneca

Indication: Limited-stage small cell lung cancer

AstraZeneca announced that the FDA has approved a new indication for Imfinzi (durvalumab) to treat patients with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed after concurrent platinum-based chemotherapy and radiation therapy (cCRT).

This approval, based on Phase III ADRIATIC study data, showed that Imfinzi significantly reduced the risk of death by 27% and extended progression-free survival by 24% compared to placebo.

Bizengri

Developing Company: Merus

Indication: Pancreatic cancer and non-small cell lung cancer with NRG1 mutations

Merus' bispecific antibody Bizengri (zenocutuzumab) has been approved by the FDA as the first and only treatment for advanced, unresectable, or metastatic pancreatic cancer and non-small cell lung cancer with NRG1 fusions.

Bizengri works by inhibiting the dimerization of HER2 and HER3, blocking the interaction between NRG1 and HER3. The approval was based on results from the eNRGy trial, which showed an objective response rate of 40% in pancreatic cancer and 33% in NSCLC patients.

These are just some of the major drugs that received FDA approval in December 2024, addressing a variety of conditions from cancer to metabolic disorders. It is always important to consult healthcare professionals for more information on how these treatments may benefit individual patients.