2024’s New FDA Drug Approvals


🌟 Discover the latest from the FDA’s CDER: 9 innovative drugs for Q1!
🧴Berdazimer (Zelsuvmi) tackles topical molluscum contagiosum with nitric oxide release.
💉Cefepime/enmetazobactam (Exblifep) combines forces for urinary tract infection relief.
💉LetibotulinumtoxinA (Letybo) smooths glabellar lines with botulinum neurotoxin.
💉Tislelizumab (Tivembra) fights esophageal squamous cell carcinoma with PD-1 mAb technology.
💊Resmetirom (Rezdiffra), a thyroid hormone receptor beta agonist, battles nonalcoholic steatohepatitis.
💊Aprocitentan (Tryvio) manages hypertension through endothelin receptor antagonism.
💊Givinostat (Duvyzat) aims at Duchenne muscular dystrophy with HDAC inhibition.
💉Sotatercept (Winrevair), an activin inhibitor, offers hope for pulmonary arterial hypertension patients.
💊Vadadustat (Vafseo) addresses CKD-related anemia in dialysis patients as an oral HIF-PH inhibitor.

Explore these groundbreaking treatments! Our appreciation to Chris De Savi for the great compilation!

Adenovirus formulation

This recent patent from our friend Tao Peng at Roquette on preserving an adenovirus vaccine with the help of cyclodextrins got us thinking about how CDs can be used in this area.


EP4338727 ADENOVIRUS FORMULATIONS (wipo.int)

In the context of vaccines, cyclodextrins can effectively encapsulate antigens, ensuring their protection from degradation and improving their immunogenicity. Cyclodextrins offer stability advantages to vaccines by preventing the degradation of labile vaccine components during storage and transportation. Furthermore, cyclodextrins can serve as adjuvants, potentiating the immune response triggered by vaccines. Their unique structure and interaction with the immune system enhance the recognition of antigens by immune cells, leading to an improved activation of both innate and adaptive immune responses. This adjuvant effect contributes to the development of robust and long-lasting immune protection against targeted pathogens. Owing to the distinctive attributes inherent to nanoparticles, their integration into vaccine formulations has assumed an imperative role. Through the encapsulation of vaccine antigens/adjuvants within cyclodextrin nanoparticles, the potency and stability of vaccines can be notably enhanced. In particular, the capacity of amphiphilic cyclodextrins to form nanoparticles through self-assembly without surfactants or co-solvents is a captivating prospect for their application as carrier systems for antigens. In conclusion, cyclodextrins present a promising platform for enhancing the efficacy and stability of vaccines. Their ability to encapsulate antigens, stabilize labile vaccine components and act as adjuvants demonstrates their potential to revolutionize vaccine formulation and delivery. Further research and development in this field will facilitate the translation of cyclodextrin-based vaccine technologies into practical and impactful immunization strategies, ultimately benefiting global health and disease prevention.

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease

Tay-Sachs disease, a progressive neurodegenerative disorder caused by mutations in the HEXA gene, lacks effective treatment. This study explores the therapeutic potential of δ-tocopherol and hydroxypropyl-β-cyclodextrin, targeting lysosomal exocytosis in Tay-Sachs models. Results indicate significant lysosomal GM2 reduction, offering hope for future treatments.

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease (hindawi.com)

Methods for the treatment of chronic kidney disease

We are happy to announce the acceptance of a great patent from CarboHyde’s collaborator, Renatus, a pharmaceutical company focused on developing cyclodextrin-based cholesterol metabolism modulators. Renatus is a pharmaceutical company whose mission is to provide safe and effective treatment options for the treatment of numerous cholesterol-driven diseases such as chronic kidney disease, atherosclerosis, and Alzheimer’s diseases. The core technology of the company allows the normalization of cholesterol homeostasis by removal of excessive and toxic cholesterol within diseased cells without significant harmful effects on normal cells.

The lead asset RN-005 is a cholesterol metabolism modulator that has proven strong therapeutic efficacy in preclinical models of both diabetic kidney disease (DKD) and focal segmental glomerulosclerosis (FSGS) wherein dysregulated cholesterol metabolism causes progressive damage and loss of function in the kidney. The gamma-cyclodextrin oligomers are effective in cholesterol metabolism enhancement, cholesterol efflux, reducing inflammatory cytokine secretion, renal clearance of cholesterol, and/or reducing albuminuria. Therefore, the gamma-cyclodextrin oligomers can be used to treat or alleviate chronic kidney disease, symptoms thereof and/or complications related to chronic kidney disease.

Espacenet – METHODS FOR THE TREATMENT OF CHRONIC KIDNEY DISEASE

Hydrogel composition for use in the treatment of articular disorders

Fascinating patent by Bernard MartelNicolas Blanchemain and colleagues presenting hydrogels on anionic cyclodextrin polymers and chitosan for use in the treatment of articular disorders. The hydrogel combines an analgesic action with a visco-supplementation (a lubricating effect).

WO2024023350 HYDROGEL COMPOSITION FOR USE IN THE TREATMENT OF ARTICULAR DISORDERS (wipo.int)

Resveratrol eye drops as well as preparation method and application

In some cases, the simplest, most straightforward ideas are the best. Why not develop an eye-drop based on cyclodextrin and resveratrol?  The resveratrol captured in an HPBCD inclusion complex has the advantages of simple preparation, short preparation period, no organic solvent residue, and no bacteriostatic agent. The lipoic acid added in the clathrate eye drops prepared by the invention cooperates with the resveratrol to play a better antioxidant effect so that the xerophthalmia is better relieved, and the treatment effect is achieved.

What to create similar solutions? Visit: www.carbohydesolutions.com – Carbohyde
Original paper: Espacenet – Resveratrol eye drops as well as preparation method and application thereof

Adamgammadex vs Sugammadex

Check out this fascinatnig discussion on next future of cyclodextrin-based antidotes and scavengers by J Robert Sneyd adn Jennifer Hunter.

Spoiler alert: Phase 3 clinical trials suggest that Adamgammadex is slightly less potent than sugammadex and has a non-inferior speed of onset. In a multicentre trial of 310 patients, there was a suggestion of a lower incidence of allergic responses and recurarisation after adamgammadex compared with sugammadex.

If you are also thinking about creating a special cyclodextrin like that, this may be useful for you: Custom synthesis of cyclodextrins – www.carbohydesolutions.com

Is adamgammadex the brother of sugammadex or the next generation of reversal agent? – British Journal of Anaesthesia (bjanaesthesia.org)

Mannose-methyl-β-cyclodextrin suppresses tumor growth by targeting both colon cancer cells and tumor-associated macrophages

We have known for a while that methylated β-cyclodextrin (MβCD) can extract cholesterol from lipid rafts and induce apoptosis in cancer cells by inhibiting activation of the PI3K-Akt-Bad pathway.

In this study from Kumamoto University (Keiichi Motoyama), MβCD with mannose (Man-MβCD) is assessed for targeting colon cancer cells expressing the mannose receptor (MR) and tumor-associated macrophages (TAM). Man-MβCD showed a significantly greater level of cellular association with colon-26 cells and M2 macrophages, and much more prominent anticancer activity than that of MβCD against MR-positive colon-26 cells. These results revealed that autophagy was the main mechanism of cell death associated with Man-MβCD. Furthermore, compared with MβCD, Man-MβCD significantly reduced tumor development following intravenous delivery to tumor-bearing mice, with no apparent side effects. Thus, Man-MβCD has the potential to be a novel anticancer drug.

If you would like to try something like this, check out this site for ideas: Custom synthesis of cyclodextrins – www.carbohydesolutions.com

Therapeutic polypseudorotaxane hydrogels

Qrons Inc. – ido merfeld propose using polyrotaxanes (which are “necklace” like molecules where the “pearls” are cyclodextrins and in this invention the “chain” is a polymer of ethylene oxide) for manufacturing a medicament for Central Nervous System. The proposed examples include piping into the brain for treatment of traumatic brain injury.
If you are interested in creating such innovative constructs from cyclodextrins, check out this site for possibilities, we can probably help: Custom synthesis of cyclodextrins – www.carbohydesolutions.com

WO2023194821 THERAPEUTIC POLYPSEUDOROTAXANE HYDROGELS (wipo.int)