In August 2021, the FDA approved a total of 4 new drugs, including 2 chemical drugs and 2 biological drugs. Let's review the new drugs approved by the FDA in August with TOSUN!

avalglucosidase alfa

On August 6, Sanofi’s avalglucosidase alfa (trade name: Nexviazyme) was approved by the US FDA for the treatment of late-onset Pompe disease for patients aged 1 and over. Previously, the FDA granted Nexviazyme breakthrough therapy qualification and fast-track qualification for the treatment of Pompe disease.

Nexviazyme can help reduce glycogen accumulation and is given by intravenous infusion every 2 weeks. The key way to transport the GAA enzyme to the intracellular lysosome is through the M6P receptor. Nexviazyme is a high-efficiency enzyme replacement therapy that can target the mannose 6-phosphate (M6P) receptor to improve cellular enzyme uptake and increase glycogen clearance in target tissues.

belzutifan

On August 13, Merck's oral hypoxia-inducible factor-2α (HIF-2α) inhibitor Welireg (belzutifan) was approved by the US FDA for the treatment of Hippel-Lindau syndrome (von Hippel-Lindau, VHL) disease Related cancers, including: renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastoma, or pancreatic neuroendocrine tumor (pNET), these patients do not require immediate surgery. Previously, the FDA granted Welireg the qualification for breakthrough therapy for this indication.

Welireg is the first HIF-2α inhibitor therapy approved by the FDA. By inhibiting HIF-2α, Welireg can reduce the transcription and expression of HIF-2α target genes related to cell proliferation, angiogenesis, and tumor growth.

difelikefalin

On August 23, Cara Therapeutics and Vifor Pharma jointly announced that the US FDA has approved the marketing of difelikefalin (Korsuva) for the treatment of moderate to severe pruritus related to hemodialysis (HD) adult chronic kidney disease (CKD-aP). This is the first and only treatment approved by the FDA to treat patients with chronic kidney disease-related pruritus. Previously, the FDA granted difelikefalin the breakthrough therapy qualification for the treatment of this indication.

difelikefalin is a first-in-class highly selective k-opioid receptor (KOR) full agonist, which can inhibit the activity of peripheral neurons that produce itching, and has an effect on other receptors (including mu or delta opioid receptors), ion channels Or the transporter has no significant activity. In addition, unlike small molecule KOR agonists, Korsuva is a small synthetic peptide that mainly activates KORs expressed by peripheral neurons (PNS) and immune cells.

On May 23, 2018, Vifor Pharma obtained the global license of Cara Therapeutics' difelikefalin in other regions except the United States, Japan, and South Korea, with a total amount of up to 540 million U.S. dollars.

TransCon Human Growth Hormone

On August 25, Ascendis Pharmaceuticals TransCon human growth hormone (lonapegsomatropin) was approved by the US FDA for the treatment of childhood growth hormone deficiency (PGHD). This is the first product approved by the US FDA to deliver growth hormone through a single injection that can be continuously released within a week.

Also approved this time is the new auto-injector and cartridge of TransCon Human Growth Hormone, which allows the drug to be stored at room temperature for up to six months after being removed from the refrigerator for the first time. Compared with daily injections, patients with weekly injections can reduce the number of injection days by 86% per year.

TransCon human growth hormone uses "temporary connection" technology to release unmodified human growth hormone in a controlled manner, maintaining the original pharmacological effects of human growth hormone. The TransConTM molecule consists of three parts: the unmodified prototype drug, the inert carrier molecule that protects the prototype drug, and the connection structure that temporarily connects the two. When the three parts are combined, the carrier molecule can inactivate the prototype drug and protect it from being eliminated by the body. When injected into the human body, the active, unmodified prototype drug will be released in a controlled manner under physiological conditions of pH and body temperature. Since the prototype drug is unmodified, it can maintain its original mechanism of action.

On March 6, 2019, Weisheng Pharmaceutical obtained the exclusive authorization of Ascendis Pharma to be responsible for the development and promotion of Ascendis Pharma's endocrine rare disease treatment programs in Greater China.

Most of the new drugs approved by the FDA this month are drugs for rare diseases. I hope that more innovative drugs will be approved to benefit the majority of patients.