Update on Trends in Emerging Therapies and Manufacturing

Single helix RNA, Epigenetics concept | Image Credit: © Artur - stock.adobe.com

From cancer to COVID-19, the middle third of 2024 saw a slew of approvals and expanded indications for new and emerging therapies worldwide. Drugs were steadily updated or introduced, and moves were also being made in outsourcing, manufacturing, acquisitions, and new partnerships. The following is a survey of the most impactful developments.

Next-generation products and strategies

With the fifth anniversary of the COVID-19 pandemic approaching, and as a summer surge increased pressure to respond, FDA on Aug. 22 announced it granted an Emergency Use Authorization for updated versions of messenger RNA (mRNA) SARS-CoV-2 vaccines made by Moderna and Pfizer (1). The vaccines are designed to primarily target the KP.2 strain of the dominant Omicron variant; however, it is likely that the major vaccine developers are already working on a formula for 2025–2026.

The COVID vaccines elevated mRNA into the mainstream lexicon, and while not foreshadowing this new round of shots, when Pharmaceutical Technology® spoke with Christian Cobaugh, PhD, CEO and founder of Vernal Biosciences, at BIO 2024 in San Diego, Calif., in June, Cobaugh addressed the landscape for the outsourcing of mRNA production in 2025 and beyond (2).

“There [are] those that just generally do GMP [good manufacturing practice] for multi different types of drug products, and then there [are] more specialty companies that were set up to make nucleic acids,” Cobaugh said in reference to mRNA service providers, pivoting to a discussion of next-generation sequencing (NGS). “NGS offers … an opportunity to go deeper and pick off multiple critical quality attributes from the same test, which, along with consolidating platforms more broadly, presents significant cost of goods savings in the long run.”

While not an emerging therapy per se, MediWound said on Aug. 15 that NexoBrid, a topically administered enzymatic therapeutic for removal of nonviable burn tissue, was granted a pediatric indication by FDA for treating deep partial and/or full-thickness thermal burns (3).

Treatments for cancer and other diseases of concern

New developments in oncology made headlines in summer 2024. FDA granted a Breakthrough Therapy designation to GSK for an antibody-drug conjugate (ADC) that targets a protein, B7-H3, considered to be a valuable target for cancer therapy (4). The investigational ADC has been under evaluation for treatment of patients with extensive-stage small-cell lung cancer.

Bristol Myers Squibb received validation of a TypeII variation application from the European Medicines Agency for an expansion of the indication for Breyanzi to include adults with relapsed or refractory follicular lymphoma. And the human epidermal growth factor receptor 2 (HER2)-directed ADC Enhertu, developed jointly by AstraZeneca and Daiichi Sankyo, got conditional approval from the National Medical Products Administration of China for treatment of adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinomas (5–6).

Read the article in the Emerging Therapies eBook.

Article details

BioPharm International®
Emerging Therapies eBook
September 2024
Pages: 28–30

Citation

When referring to this article, please cite it as Lavery, P. Update on Trends in Emerging Therapies and Manufacturing. BioPharm International Emerging Therapies eBook, September 2024.