22 May “FDA Decisions, Clinical Trials and Approval Milestones: Stay on top of recent updates in the Pharmaceutical Industry”

The pharmaceutical and healthcare industry has been in the spotlight in recent days with a number of important developments. We’ve brought you the most important updates so you can stay up to date with the latest news!

This week has been marked by important developments in the pharmaceutical and healthcare sectors, highlighting the dynamic view of medical advancements and regulatory decisions. From the US FDA’s delayed decision on Moderna’s RSV vaccine to Novo Nordisk’s initiation of a groundbreaking trial for alcohol-related liver disease, these updates highlight significant improvements and setbacks in the industry. Additionally, Roche’s FDA approval for a revolutionary HPV self-testing method, Lupin’s green light for Midostaurin Capsules reflect ongoing progress in addressing critical health issues and a big promise for cancer vaccines.

Here’s a closer look at these key news and their implications for the future of medicine.

The US FDA has pushed back its decision on Moderna’s RSV vaccine until the end of May.

May 10th, Moderna, based in Cambridge, Massachusetts, faced a setback as the FDA (The United States Food and Drug Administration) delayed its decision on the approval of the RSV (Respiratory Syncytial Virus) vaccine until month-end due to “administrative constraints.” This prolongs the anticipation for Moderna’s second approved vaccine. However, the company remains aligned with the CDC’s review on June 26th-27th. The CDC (The Centers for Disease Control and Prevention) panel is expected to vote on usage and target population recommendations, critical for the vaccine’s commercialization.

This delay represents a frustrating step, but Moderna remains confident that with CDC support and a positive decision in late May, the RSV vaccine can still be made available to the public. The company maintains its commitment to public health and the fight against serious respiratory diseases.

Novo Nordisk Initiates Phase 2 Trial Investigating Ozempic and Two Other Drugs for Alcohol-Related Liver Disease.

On May 15th, Novo Nordisk embarked on a phase 2 trial to assess the potential of Ozempic’s active ingredient (semaglutide, which helps to regulate blood sugar levels) along with another experimental therapy, as treatments for alcohol-related liver disease. The trial aims to evaluate their effects on liver damage and alcohol use, with the third drug targeting the fibroblast growth factor 21 hormone (FGF21, plays a significant role in regulating metabolism, including glucose and lipid metabolism) . The study, set to begin on May 20 and conclude by September 2025, will be conducted across 12 U.S. states and 13 other countries.

This trial has the potential to be a considerable advance in the treatment of alcohol-related liver disease. If successful, the study could pave the way for new therapies that significantly improve patients’ quality of life.

Roche announces FDA approval of HPV self-testing in the United States.

On the same day, May 15th,  Roche’s breakthrough human papillomavirus (HPV) self-collection option received FDA approval, revolutionizing HPV screening. This innovative solution empowers individuals to conveniently collect their own vaginal sample at healthcare facilities for HPV testing. The samples are analyzed using Roche’s cobas® molecular instrument (a versatile platform used for performing molecular diagnostics), enabling detection of 14 high-risk HPV genotypes, including HPV 16 and HPV 18. With this milestone, Roche aims to accelerate progress towards the WHO’s ambitious goal of eliminating cervical cancer by 2030.”

This approval represents a significant advancement in HPV screening, allowing greater access and convenience for early detection. Roche’s innovation has the potential to accelerate progress towards the WHO’s ambitious goal of eliminating cervical cancer by 2030.

FDA Approval: Lupin’s Midostaurin Capsules Greenlit for Treatment Milestone.

One day after the two previously mentioned FDA approvals, May 16th, Lupin, a global pharmaceutical company, was granted approval by the United States Food and Drug Administration (U.S. FDA) for its Midostaurin Capsules (a medicine that targets proteins in cancer cells and stops the cancer cells from growing). These capsules, each containing 25 mg of midostaurin, are now authorized as a generic equivalent to Rydapt® Capsules, 25 mg, produced by Novartis Pharmaceuticals Corporation. Midostaurin Capsules are prescribed for the treatment of aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell leukemia (MCL). With estimated annual sales of USD 75 million in the U.S. (according to IQVIA MAT March 2024), this approval signifies a significant milestone for Lupin.

The approval of Lupine’s Midostaurin Capsules underscores continued progress in providing essential treatments for rare and serious diseases. It also strengthens Lupine’s position in the global pharmaceutical market, through its commitment to innovation and accessibility in the treatment of complex conditions.

The Promise of Cancer Vaccines

On May 20th, Cancer, the world’s second leading cause of death after cardiovascular diseases, still lacks a cure. However, hope is on the horizon as three vaccines for specific types of skin and lung cancer have progressed to the final phase of clinical trials. If successful, these vaccines could be available within three to eleven years, offering new avenues for cancer treatment and relapse prevention.

Unlike traditional vaccines that prevent disease, these innovative vaccines aim to cure cancer or prevent its recurrence by targeting the unique genetic mutations in each patient’s tumor cells. Two of these vaccines, developed by Moderna and Merck, are personalized neoantigen therapies (to train a patient’s immune cells to recognize the cancer fingerprint proteins and launch an immune response against the cancer cells). They have shown significant promise in phase II trials, particularly when combined with immunotherapy, in preventing relapses of melanoma and non-small cell lung cancer.

Currently, these vaccines are being tested on a larger group of patients in phase III trials, with results anticipated by 2030 for melanoma (a disease characterized by the formation of malignant cells (cancer) from melanocytes (cells that give color to the skin)) and 2035 for lung cancer. Additionally, OSE Immunotherapeutics’ vaccine for advanced non-small cell lung cancer, Tedopi (a novel T-cell epitope-based cancer vaccine targeting five tumor-associated antigens, an activating and differentiated off-the-shelf immunotherapy expanding tumor specific T-lymphocytes in HLA-A2 cancer patients.), is also showing promise and may be available by 2027.

Other vaccines targeting pancreatic and colon cancer are in earlier trial phases, utilizing similar personalized approaches. Advances in RNA technology (a technology that encompasses a variety of techniques and applications that involve the manipulation and use of RNA molecules for research, diagnostic, and therapeutic purposes), including mRNA and siRNA therapies (innovative approaches in the field of molecular medicine that leverage the properties of RNA to treat a variety of diseases), are contributing to the development of more effective cancer treatments.

Artificial intelligence has been an ally, further enhancing these therapies by identifying genetic mutations and managing personalized treatment data. Despite challenges in delivering personalized treatments cost-effectively and promptly, these advancements signify substantial progress in the fight against cancer.

These achievements indicate a renewed commitment to more effective and personalized treatments, offering hope for the future of medicine and global health.

Stay tuned for more news and updates.

See you next week!

DOit Team