The pharmaceutical industry is set for a major shake-up in 2024. New approaches like drug antibody combos as Antibody-drug conjugate (ADC) or use of CRISPR gene editing technology are creating treatments for patients who have few options left. This year is expected to see a wave of 10 brand new drugs and therapies hit the market, targeting diseases like breast cancer, blood clotting disorders, and chronic illnesses.[1]
Eylea HD (aflibercept), a high-dose injection, has been approved by the FDA (US) to treat wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retinopathy (DR). Regeneron and Bayer collaborated to develop Eylea HD. Regeneron holds exclusive rights to Eylea and Eylea HD in the United States, while Bayer has licensed exclusive marketing rights outside the U.S. The FDA based its approval on results from two clinical trials (PULSAR and PHOTON) that compared Eylea HD to the standard Eylea (aflibercept) injection. [2]
Calliditas Therapeutics developed a new drug called Nefecon, a more targeted and safer version of the steroid budesonide. Nefecon treats a kidney disease called primary IgA nephropathy. It helps reduce protein in the urine and slow down kidney decline. Nefecon has different brand names depending on the region: Tarpeyo used in the United States (approved by FDA) & Kinpeygo used in the European Union (conditionally approved). Their partnerships with STADA Arzneimittel AG that sells Kinpeygo in Europe for Calliditas & Everest Medicines that sells Nefecon in China and other Asian countries. The clinical trial Nefigard that led to FDA approval in the US. It was a randomized, double-blind study with 365 participants who received Nefecon (16mg daily) for 9 months. [3,4]
Datopotamab deruxtecan (Dato-DXd) developed by AstraZeneca and Daiichi Sankyo, a drug being investigated as a treatment for a specific type of breast cancer: previously treated, metastatic hormone receptor-positive (HR-positive), HER2-negative breast cancer. The FDA has considered approval to Dato-DXd based on a successful clinical trial (TROPION-Breast01). This trial compared Dato-DXd to standard chemotherapy in patients with this type of breast cancer. Dato-DXd is ADC drug type and it works by targeting a protein called TROP2 on cancer cells and then delivering a cell-killing toxin. [5]
Efanesoctocog alfa (Altuviiio) is a new medication for Hemophilia A, developed by Sanofi and Sobi. Unlike other Hemophilia A treatments, Altuviiio works independently of von Willebrand factor, making it a potential option for more patients. This medication is approved for both adults and children with Hemophilia A. In a clinical trial (XTEND-1), once weekly injections of Altuviiio proved effective in preventing bleeding episodes in people with severe Hemophilia A. [6]
Ensifentrine is a new drug being developed by Verona Pharma to treat chronic obstructive pulmonary disease (COPD). It works differently from other COPD medications by combining two effects in one medicine: Bronchodilation: relaxes muscles around the airways to ease breathing; and Anti-inflammatory: reduces inflammation in the airways. Ensifentrine is inhaled as a mist (nebulizer) and is currently undergoing Phase 3 clinical trials to see how effective it is for COPD maintenance treatment. These trials are called ENHANCE (Ensifentrine as a Novel Inhaled Nebulized COPD Therapy). [7]
Casgevy (exa-cel) is a groundbreaking new gene therapy treatment developed by CRISPR Therapeutics and Vertex Pharmaceuticals. It's the first-ever treatment approved anywhere in the world that uses CRISPR gene-editing technology. Casgevy is designed to help patients with two serious blood diseases: sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). It's intended for patients aged 12 and older who meet certain criteria, including having no suitable bone marrow donor. Early clinical trials showed promise, with many patients experiencing significant improvement in their condition. [8]
Lyfgenia (lovo-cel) is a new gene therapy from Bluebird Bio for sickle cell disease in patients 12 and older. It's a one-ti...
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