2024 Medical Breakthroughs: New Hope to Patients

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-time treatment that works by modifying a patient's own stem cells to produce healthy adult hemoglobin. This reduces the risk of sickle cells forming and causing painful episodes (vaso-occlusive events). Key points associated with this drug are it's being approved by the FDA for patients with a history of these painful episodes, early trials showed success in reducing them, common side effects include mouth sores, low blood cell counts, and a long-term safety is being monitored due to potential risks. ^[9]

Eli Lilly received FDA approval for Omvoh (mirikizumab), a new treatment for moderately to severely active ulcerative colitis (UC) in adults. This is the first medication of its kind to target a specific protein (IL-23p19) involved in UC. Omvoh is designed to help patients who haven't responded well to other UC treatments.  Early trials (LUCENT program) showed promise in reducing a major symptom of UC: bowel urgency.  Omvoh is given as an infusion or injection. ^[1]

The Janssen Pharmaceutical Companies of Johnson & Johnson developed AKEEGA™ is the first single-pill treatment option for men with a specific type of advanced prostate cancer. This type of cancer is called BRCA-positive metastatic castration-resistant prostate cancer (mCRPC). AKEEGA™ combines two medications (niraparib and abiraterone acetate) into one tablet for easier use. Clinical trials (including the large MAGNITUDE study) showed AKEEGA™ to be effective in extending the time before the cancer worsens compared to standard treatment. FDA Approves another J&J's Talvey™ for Heavily Treated Multiple Myeloma. Talvey™ is a first-of-its-kind treatment for patients with multiple myeloma who haven't responded well to other therapies. It boasts a high response rate (over 70%) and works by targeting specific proteins on myeloma cells and immune cells. Talvey™ is a flexible treatment option, offered as a weekly or bi-weekly injection. ^[10,11]

In conclusion, 2024 is shaping up to be a transformative year for medicine with a wave of innovative treatments for various conditions. This includes breakthroughs in gene therapy, targeted therapies for cancers, and combination drugs offering new hope for patients with limited options. The focus on unmet medical needs and utilizing novel technologies like antibody-drug conjugates and CRISPR gene editing is a promising trend for the future of healthcare. 

References:

1.Diana Spencer Thirteen potential blockbuster drugs to watch in 2024 Drug Discovery World Published on 9 January 2024; Accessed on 8 April 2024.   URL: https://www.ddw-online.com/thirteen-potential-blockbuster-drugs-to-watch-in-2024 27823-202401/

2. Regeneron EYLEA HD (aflibercept) Injection 8 mg Approved by FDA for Treatment of Wet Age-related Macular Degeneration (wAMD), Diabetic Macular Edema (DME) and Diabetic Retinopathy (DR) Published on August 18, 2023 at 6:35 PM EDT; Accessed on 8 April 2024.   URL:https://investor.regeneron.com/news-releases/news-release-details/eylea-hd aflibercept-injection-8-mg-approved-fda-treatment-wet

3. PDF report Calliditas Therapeutics Stockholm, Sweden 'Calliditas Receives Notice of Allowance for United States Patent Application Covering TARPEYO®', report dated December 11, 2023.   Report available online via URL link: https://mb.cision.com/Main/16574/3891581/2485500.pdf

4. ClinicalTrials.gov Identifier: NCT03643965 Efficacy and Safety of Nefecon in Patients With Primary IgA (Immunoglobulin A) Nephropathy (Nefigard), Published on: August 23, 2018 Updated on: July 18, 2023; Accessed on: April 8, 2024.   URL: https://classic.clinicaltrials.gov/ct2/show/NCT03643965

5. Adrian Kemp AstraZeneca PLC 'Datopotamab deruxtecan Biologics License Application accepted in the US for patients with previously treated metastatic HR-positive, HER2 negative breast cancer' Published on: 2 April 2024; Accessed on : 8 April 2024. URL: https://www.astrazeneca.com/media-centre/press-releases/2024/fda-accepts-dato-dxd bla-for-breast-cancer.html

6. Keam SJ. Efanesoctocog Alfa: First Approval. Drugs. 2023 May;83(7):633-638. doi: 10.1007/s40265-023-01866-9. Erratum in: Drugs. 2023 Jul;83(10):953. PMID: 37022666; PMCID: PMC10238336.

7. URL: https://www.veronapharma.com/media/verona-pharma-announces-ensifentrine meets-primary-and-key 

8. VERTEX News & Events Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia Published on: Nov 16, 2023, Accessed on: April 8, 2024.  

URL : https://investors.vrtx.com/news-releases/news-release-details/vertex-and-crispr therapeutics-announce-authorization-first 

9. Kadeja Johnson PHACILITATE bluebird bio Announces FDA Approval of LYFGENIA for Sickle Cell Disease but Faces Concerns Published on: 12 December 2023, Accessed on: 8 April 2024.   URL: https://www.phacilitate.com/news-bluebird-bio-announces-fda-approval-of-lyfgenia for-sickle-cell-disease-but-faces-concerns/ 

10. J&J Press Release/Pharmaceuticals U.S. FDA Approves AKEEGA™ (Niraparib and Abiraterone Acetate), the First-And-Only Dual Action Tablet for the Treatment of Patients with BRCA-Positive Metastatic Castration-Resistant Prostate Cancer, Published on: August 11, 2023; Accessed on: April 8, 2024.  

URL: https://www.jnj.com/media-center/press-releases/u-s-fda-approves-akeega-niraparib and-abiraterone-acetate-the-first-and-only-dual-action-tablet-for-the-treatment-of-patients with-brca-positive-metastatic-castration-resistant-prostate-cancer

11. J&J Press Release/Pharmaceuticals U.S. FDA Approves TALVEY™ (talquetamab-tgvs), a First-in-Class Bispecific Therapy for the Treatment of Patients with Heavily Pretreated Multiple Myeloma, Published on: August 10, 2023; Accessed on: April 8, 2024. URL: https://www.jnj.com/media-center/press-releases/u-s-fda-approves-talvey-talquetamab tgvs-a-first-in-class-bispecific-therapy-for-the-treatment-of-patients-with-heavily-pretreated multiple-myeloma

About the Author:

Shruti  Talashi    

Ms.  Shruti  Talashi  boasts a dual mastery of lab research and writing.  Her doctoral study outcome as M.Phil in biomedical science while studying  breast cancer and an extraordinary masters degrees dissertation work on  exploring role of Gal-lectin in cancer metastasis fuels her extensive  research interests. She has gained few publication in journals. Bridging the  science-public gap is her passion, aided by expertise in diverse techniques.  From oncology to antibiotic/drugs production, she's led and managed complex  projects, even clinical trials. Now, as a freelance Content Coordinator for  Sinoexpo Pharmasource.com, her industry knowledge shines through valuable  insights on cutting-edge topics like GMP, QbD, and biofoundry.