TOP Pharma & Biotech News #8?? August Edition

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In August, explore key insights from the FDA's workshop on AI in drug development, discover the latest trends and technologies in the growing cell and gene therapy market, and learn about the FDA's approval of the first nasal spray for anaphylaxis.

A full-day training titled "Artificial Intelligence in Drug and Biological Product Development" was held on August 6, 2024, by the FDA and the Clinical Trials Transformation Initiative (CTTI). Four panels featured speakers from the FDA, academia, lobbying groups, and the business sector, discussing AI in drug development. Most panelists called for clearer government regulations and more resources for AI development, particularly through public-private partnerships. They also emphasized the need for public education on AI’s role in drug creation to build trust and foster innovation.

Morgan Hanger, CTTI's Executive Director, opened the event, highlighting AI’s transformative impact on drug research, focusing on clinical development and study design improvements. The keynote by Dr. Patrizia Cavazzoni, Director of FDA’s Center for Drug Evaluation and Research (CDER), addressed AI’s integration into drug creation, noting the need for clearer industry guidance. The FDA is developing risk-based guidelines to provide greater predictability and encourage innovation.

Session 1 emphasized interdisciplinary collaboration in AI model development for drug research, advocating for showcasing AI successes to build trust and understanding. The speakers urged support for AI integration rather than resistance due to unknown risks.

Session 2 addressed data challenges in drug development, stressing the importance of research-ready datasets and the potential of public-private partnerships. The speakers also discussed AI bias, urging the need for controls to monitor and mitigate bias throughout AI’s lifecycle. They called for increased data transparency and availability, along with FDA guidelines to support this.

Session 3 focused on AI model explainability, essential for regulatory trust and industry investment. The panel suggested the FDA highlight successful AI applications to boost confidence and called for a clear regulatory framework to reduce uncertainties and encourage innovation.

Sessions 4 and 5 reiterated the need for clearer regulations, government collaboration, and global alignment on AI standards. The panelists likened AI’s current state to the early days of the internet, urging significant federal investment to set rules and guidelines. They stressed the importance of global cooperation on AI terms and standards to ensure consistency and accessibility.

Jacqueline Corrigan-Curay, Principal Deputy Center Director of CDER, concluded the workshop, echoing the potential of AI highlighted by Dr. Cavazzoni. The event underscored the FDA's commitment to leveraging AI to enhance drug research and safety, with a strong emphasis on the need for a stable legal framework and development path for AI in drug development.

Source: WORLD PHARMA TODAY

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In 2023, the cell and gene therapy market was valued at USD 18.8 billion, with a projected annual growth rate of 15.66%, expected to reach USD 60.1 billion by 2032.

The market’s expansion is driven by regulatory changes, continuous technological advancements, substantial investments, increasing healthcare costs, strong government support in developed countries, rapid infrastructure development, growing patient awareness, extensive R&D activities, and strategic partnerships between academia and industry.

Regulatory improvements are a significant driver, with agencies like the FDA and EMA streamlining the development and approval of new therapies through detailed guidelines and accelerated approval processes. Programs such as the Priority Medicines (PRIME) in Europe and the Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. have expedited the review of treatments addressing unmet medical needs. These regulatory initiatives have reduced the time and cost required to bring cell and gene therapies to market, fostering increased investment and research.

Harmonization of regulations ensures that therapies meet high safety and efficacy standards, facilitating global market entry. Regulatory bodies are increasingly collaborating with stakeholders through public consultations and advisory groups to ensure that new regulations are grounded in scientific and commercial realities.

There are currently over 3,800 gene therapy studies listed on clinicaltrials.gov , with more than 1,000 actively recruiting. Gene therapy Investigational New Drug (IND) applications have surged, with the FDA expecting to approve up to 20 gene therapies annually by 2025, supported by a robust pipeline of clinical trials.

Technological advancements, particularly in gene editing technologies like CRISPR-Cas9, have revolutionized the market by enabling precise genetic modifications. These innovations have paved the way for targeted and personalized treatments, offering potential cures for previously untreatable genetic disorders. Advanced viral vectors and site-specific gene editing technologies have further enhanced the efficacy and safety of therapies, such as hematopoietic stem/progenitor cell gene therapy (HSPC-GT), which shows promising clinical outcomes.

Improvements in cell engineering, exemplified by the success of CAR-T cell therapy in treating certain cancers, have significantly boosted the effectiveness of cell-based treatments. Additionally, innovations in manufacturing, such as automated cell culture systems, have increased the scalability and quality control of cell therapies, addressing a major industry challenge. The integration of artificial intelligence (AI) and machine learning (ML) into drug discovery has also accelerated the identification of therapeutic targets and optimized clinical trial designs, making the development process faster and more cost-effective.

The market has also benefited from a surge in investments and funding from venture capital firms, pharmaceutical companies, and government bodies. This influx of capital has been crucial for advancing R&D, funding clinical trials, and facilitating the commercialization of new therapies. Strategic collaborations between pharmaceutical giants and biotech companies have accelerated innovation by combining resources and expertise, leading to the faster development of new treatments. Government funding and initiatives have also played a vital role in supporting research, particularly for rare diseases.

The rise in IPOs and mergers and acquisitions (M&A) within the industry reflects growing investor confidence, providing companies with the financial resources needed to expand operations and advance research projects. This strong financial backing not only ensures the sustainability of companies developing these therapies but also fosters a competitive environment that encourages innovation and accelerates the introduction of new treatments.

Leading companies in the global cell and gene therapy market, including Bristol-Myers Squibb, Gilead Science, Pfizer, and Spark Therapeutics, are scaling up production, forming strategic partnerships, and securing regulatory approvals. Recent FDA approvals, such as BLINCYTO? for treating B-ALL and QALSODY? for SOD1-ALS, highlight the ongoing innovation and impact of these therapies.

Geographically, the U.S. and Canada dominate the North American market, driven by a robust healthcare system, significant R&D investments, and a favorable regulatory environment. Europe, led by countries like Germany and the UK, benefits from strong government support, academic-industry collaborations, and a unified regulatory framework provided by the EMA. The Asia-Pacific region is experiencing rapid growth due to increased healthcare spending and technological advancements, with China and Japan leading the way. Latin America and the Middle East and Africa are also seeing growth, driven by improving healthcare infrastructure and increased investment in biotechnology.

Overall, cell and gene therapies are transforming the treatment landscape for a wide range of diseases, particularly in oncology and genetic disorders. These therapies offer innovative and potentially curative solutions, driving global market growth and reshaping patient outcomes.

Source: WORLD PHARMA TODAY

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The U.S. Food and Drug Administration approved neffy (epinephrine nasal spray) for emergency treatment of life-threatening allergic reactions (anaphylaxis) in adults and children weighing at least 30 kilograms (66 pounds).

"Neffy is the first non-injection epinephrine product for anaphylaxis treatment, addressing concerns about injection fear, particularly in children," said Kelly Stone, MD, PhD, Associate Director at the FDA’s Center for Drug Evaluation and Research. "This nasal spray may reduce treatment barriers and meet an unmet need."

Anaphylaxis is a severe, life-threatening allergic reaction affecting multiple body parts and requiring immediate medical attention. Common triggers include foods, medications, and insect stings. Symptoms, such as hives, swelling, difficulty breathing, and loss of consciousness, occur within minutes. Epinephrine, the only life-saving treatment, was previously available only by injection.

Neffy's approval is based on studies comparing epinephrine levels in the blood after using neffy versus injection products. Results showed similar effectiveness, including necessary increases in blood pressure and heart rate.

Neffy is a single-dose nasal spray, with a second dose possible if symptoms don't improve. Patients should seek emergency medical care for further monitoring.

Neffy carries warnings for patients with nasal conditions and certain coexisting conditions. Common side effects include throat irritation, nasal discomfort, headache, and nausea.

The FDA granted neffy Fast Track designation and approved it for ARS Pharmaceuticals.

Source: WORLD PHARMA NEWS

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