New Drug Spotlight | September: NMPA Approves Two Rare Tumor Therapies, FDA Clears Multiple Hematologic Oncology Innovations

Highlights

CSPC's New Class 1 Drug Narusobanumab Approved for Marketing by NMPA

Multiple Imported New Drugs Approved in China

FDA Approves First Innovative Drug for Stem Cell Mobilization in Multiple Myeloma

FDA Approves New Drug for High-Risk Myelofibrosis Treatment

Which new drugs were approved for marketing in September? For more details, please see the following.

01

Innovative drugs approved for marketing by NMPA

In September, the NMPA approved 6 new drugs, including 2 domestically produced new drugs and 4 imported new drugs. For details, see the table below:

Table 1. New drugs approved by NMPA in September 2023

Data Source: NMPA Official Website

Naruzumab

On September 6, 2023, Jinmate Biological Technology (Shanghai), a subsidiary of CSPC, received conditional approval from the NMPA through the priority review and approval process for its Category 1 new drug Narusobimab Injection (trade name: Jinlisheng) to treat adult patients with giant cell tumor of bone that is inoperable or where surgical resection may lead to severe functional impairment.

Giant Cell Tumor of Bone is a relatively rare benign bone tumor but exhibits local invasiveness and osteolytic characteristics, commonly occurring in young adults. The main clinical manifestations include dull pain, fractures, functional limitation, as well as swelling and masses. The annual incidence rate of Giant Cell Tumor of Bone in China is approximately 1.49-2.57 cases per million.

Naruluximab is the world's first approved IgG4 subtype fully human anti-RANKL monoclonal antibody. This product is administered via subcutaneous injection and works by blocking the binding of RANKL to the RANK receptor on the membranes of osteoclast precursor cells, osteoclasts, and osteoclast-like giant cells. This inhibits the RANKL-RANK signaling pathway-mediated differentiation, maturation, and functional activity of these cells.

Narusobimab, currently under development for the treatment of bone metastases from tumors and osteoporosis, has an optimized structure compared to denosumab (IgG2 subtype) targeting the same receptor, enhancing affinity and simplifying the production process.

Table 2. Imported new drugs approved by NMPA in September 2023

Data Source: NMPA Official Website

Mitotane Tablets

On September 5, 2023, the NMPA approved the marketing of mitotane tablets by HRA Pharma Rare Diseases for the treatment of adrenocortical carcinoma (ACC). Previously, the CDE had included the marketing application of mitotane tablets in the priority review.

The adrenal glands, located directly above the kidneys, produce various hormones, including cortisol, aldosterone, and male sex hormones. Tumors in these glands are very common, but adrenocortical carcinoma is a rare and aggressive cancer that grows on the outer layer of the gland. Adrenocortical carcinoma can occur at any age but is more commonly found in children under 10 years old and adults between 40-50 years old. This is the disease that Shi Yonggang, the author of "Patients Without Borders," was diagnosed with.

Mitotane is an adrenocorticolytic agent that inhibits the adrenal cortex from producing corticosteroid hormones. The drug was approved in the United States in 1970 for the treatment of adrenocortical carcinoma. In 2018 and 2019, China imported some of the drug through a temporary import pathway (without clinical trials). This time, it has finally been approved by the NMPA for marketing in China, bringing hope to patients with adrenocortical carcinoma in China.

Fluticasone/Formoterol Inhalation Aerosol

Flutiform (Fluticasone and Formoterol Inhalation Aerosol) is a combination drug that mixes fluticasone and formoterol in a single inhaler, which can take effect rapidly within 1 to 3 minutes. It has previously been approved for the treatment of asthma in Hong Kong, China (brand name: Flutiform).

Flutiform utilizes the traditional inhaler design commonly used by asthma patients, with a dose display window on the inhaler for easy checking of doses. This helps enhance patients' habit of taking medication on time and more effectively control asthma.

Ezetimibe Atorvastatin Calcium Tablets (I)/(II)

Ezetimibe and Atorvastatin Calcium Tablets (I)/(II) submitted by Organon, trade name: Yilituo, have been granted marketing approval by the NMPA for the treatment of hypercholesterolemia and homozygous familial hypercholesterolemia (HoFH).

Ezetimibe Atorvastatin Calcium Tablets are a fixed-dose combination of ezetimibe and atorvastatin, originally developed by Schering-Plough, and currently held by Merck. In 2013, Ezetimibe Atorvastatin Calcium Tablets were approved by the U.S. FDA for treating elevated LDL-C in patients with primary or mixed hyperlipidemia, and for reducing cholesterol levels in patients with homozygous familial hypercholesterolemia (FH). Ten years later, the drug was approved in China, offering a new treatment option for patients with dyslipidemia who require combination therapy.

Notably, in the same month, the first domestically produced ezetimibe and atorvastatin calcium tablet (I)/(II) applied for production registration by Jiangxi Shimei Pharmaceutical Co., Ltd. was accepted by the CDE and entered the review stage.

Erenumab Injection

On September 21, 2023, the NMPA approved Novartis' Erenumab Injection for marketing in China, intended for the prevention of migraine in adults. This medication requires only one injection per month and is easy to administer, with patients able to complete the dosing within 15 seconds.

Erenumab Injection, developed by Novartis in collaboration with Amgen, is the world's first fully human monoclonal antibody that specifically targets the CGRP receptor. The drug works by blocking the CGRP receptor to prevent migraines and exhibits low immunogenicity.

The drug was approved by the FDA in May 2018 for preventive treatment of migraine in adults and launched in the EU under the name Erenumab in July of the same year. In July 2022, Novartis announced that Erenumab had been introduced to the Guangdong-Hong Kong-Macao Greater Bay Area, and in September this year, it received NMPA approval for marketing in China.

02

New Drug Approved by the U.S. FDA

In September, the FDA's Center for Drug Evaluation and Research (CDER) approved eight new drugs, including three new molecular entities APHEXDA, OJJAARA, and EXXUA, one new drug combination (DOLUTEGRAVIR; TENOFOVIR DISOPROXIL FUMARATE; LAMIVUDINE), two new formulations CYCLOPHOSPHAMID and LIKMEZ, one new dosage form RYZUMVI, and one drug XALKORI.

Table 3. New drugs approved by the US FDA in September 2023

Data Source: FDA official website

EXXUA

EXXUA (gepirone) is an oral serotonin (5HT) 1A receptor agonist developed by Fabre-Kramer Pharmaceuticals, Inc. for the treatment of major depressive disorder (MDD) and other psychological disorders.

Depression is one of the most common mental disorders, with approximately 280 million patients worldwide. Meanwhile, nearly two-thirds of depression patients fail to achieve adequate relief from existing therapies. The main drugs currently available on the market for treating depression include: fluoxetine (Prozac), paroxetine, sertraline, fluvoxamine, and citalopram. The approval of innovative drugs will provide new options for patients.

OJJAARA

OJJAARA (momelotinib) is a once-daily oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor approved by the FDA for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis and secondary myelofibrosis (post-polycythemia vera and post-essential thrombocythemia) in adult patients with anemia.

Myelofibrosis is a rare blood cancer primarily caused by dysregulated JAK-STAT signaling, with main symptoms including anemia, systemic symptoms, and splenomegaly.

OJJAARA has a unique mechanism of action, inhibiting three key signaling pathways: Activin A receptor type I (ACVR1), JAK1, and JAK2. The drug was developed by Sierra Oncology and, in July 2022, GlaxoSmithKline (GSK) completed the acquisition of Sierra Oncology, thereby obtaining the development rights to OJJAARA. According to GSK's press release, OJJAARA is the only approved drug for newly diagnosed and previously treated anemic myelofibrosis patients, addressing the main symptoms of myelofibrosis—namely anemia, systemic symptoms, and splenomegaly.

APHEXDA

On September 8, 2023, the FDA approved APHEXDA in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.

Multiple Myeloma is the Second Most Common Hematologic MalignancyThe American Cancer Society states that in 2023, more than 35,000 people in the United States are expected to be diagnosed with multiple myeloma, and nearly 13,000 will die from the disease. Autologous stem cell transplantation is a part of the standard care for multiple myeloma, which can extend patients' survival. In the U.S., approximately 8,000 patients undergo autologous stem cell transplantation each year. The success of autologous stem cell transplantation depends on the adequate mobilization of stem cells during the treatment process.

APHEXDA, developed by BioLineRx, is a CXCR4 inhibitor administered via subcutaneous injection. It is the first innovative stem cell mobilization drug approved in the U.S. for the treatment of multiple myeloma in a decade and also the first product approved for BioLineRx.

In October, which new drugs will be approved? Follow the Boyao WeChat Official Account, and our editor will keep you updated with continuous coverage~

Editor: Baiji

Disclaimer: The views expressed in this article are solely those of the author and do not represent the position of Pharma Intelligence. We welcome discussions and additional insights in the comment section; for reprints, please be sure to credit the author and source. If there are any issues regarding the content, copyright, or other aspects of the work, please leave a message on this platform, and we will address it promptly.