Received orphan drug recognition! Shanghai Pharmaceutical SPH4336 breakthrough liposarcoma

On March 28, Shanghai Pharmaceutical Group Co., Ltd. announced that its self-developed SPH4336 for the indication of liposarcoma has been granted orphan drug status by the US FDA.

Received orphan drug status

SPH4336 has great potential

SPH4336 is an oral small molecule inhibitor independently developed by Shanghai Pharmaceutical, targeting CDK4 and CDK6. According to the data, SPH4336 is currently under study for several indications, including advanced solid tumor, liposarcoma, and metastatic breast cancer.

This time, SPH4336 has been granted orphan drug designation by the FDA for liposarcoma. According to the announcement, no product with the same target has been approved by the FDA for the treatment of liposarcoma. Previously, SPH4336 showed significant antitumor effects in nude mouse subcutaneous graft models of clinically patient-derived liposarcoma PDX.

Therefore, SPH4336 has great potential in the treatment of liposarcoma.

In addition, SPH4336 is also being tested in combination with other drugs, according to Pharmacointelligence. Not long ago, Shanghai Pharmaceutical announced to cooperate with Kangfang Pharmaceutical to jointly develop SPH4336 tablets and Kaitanil combination therapy for highly differentiated liposarcoma (WDLS)/dedifferentiated liposarcoma (DDLS) and other tumor indications. The combination of SPH4336 and letrozole in the treatment of HR+/ HER2-metastatic breast cancer and breast cancer has been carried out to phase III clinical trials...

It can be seen that Shanghai Pharmaceutical has high hopes for SPH4336; And from the current clinical manifestations, SPH4336 also lived up to its expectations.

How new drugs break through,

By policy alone?

According to Shanghai Pharmaceutical, SPH4336 has been approved as an orphan drug by the US FDA this time, which can accelerate the progress of clinical trials and marketing registration.

FDA orphan drug designation is only used for diseases that have fewer than 200,000 cases in the United States, or that have more than 200,000 cases if the number of cases is larger than expected. According to incomplete statistics, nearly 100 indications of drugs developed by domestic companies have been approved as FDA orphan drugs.

One of the most famous is Beigene's Zbrutinib, which has been approved as an orphan drug for 6 indications, including mantle cell lymphoma, Fahrenheit macroglobulinemia and chronic lymphocytic leukemia. In addition, Asom's APG-2575 tablets have also been granted orphan drug status in 5 indications, including refractory chronic lymphocytic leukemia CLL, multiple myeloma and Warmulobulinemia.

Zbutinib went on sale in China in 2020 and was quickly covered by health insurance; Under the medical insurance drug and various strong indications, its sales are very substantial. For reference below, it is estimated that 500 million yuan has been reached in 2022.

Zebutinib has a number of orphan drug approvals and is supported by the national Health insurance. Therefore, it may be too early to look at Zebtinib to predict the successful launch of SPH4336; However, the potential SPH4336 is full of endless possibilities.

Of course, according to the regulations, before the FDA approval of SPH4336, if other identical drugs with the same indication are first approved, the program must be further demonstrated to be clinically superior, otherwise the policy support enjoyed as an orphan drug will be lost.

From this perspective, SPH4336 has a long way to go.

summary

Globally, orphan drug certification, breakthrough treatment certification and other policies encourage and support drug development, and play a great role in promoting the development of the pharmaceutical industry. In recent years, many drugs developed by domestic pharmaceutical companies have been recognized as FDA orphan drugs. It can be seen that the domestic pharmaceutical industry is also booming!