Orphan Drugs


In 2018, the drug racemic-3-butylphthalide, developed by CSPC for the treatment of amyotrophic lateral sclerosis (ALS), obtained orphan drug qualification awarded by FDA.

ALS is also called motor neurone disease. It is a rare disease, more common in men between 40 and 50 years old. The cause is still unknown. Some cases are believed to be related to heredity and genetic defects. Motor nerve cells undergoing progressive degeneration causes muscle weakness and atrophy in limbs, trunk, chest and abdomen, as well as speech, deglutition and respiratory function decrease, which will finally lead to respiratory failure and death.

Butylphthalide Soft Capsules developed by CSPC obtained production approval granted by China Food and Drug Administration in 2005, and it is for the treatment of mild and moderate acute ischemic stroke.

2.mitoxantrone hydrochloride liposome injection

In 2018, FDA granted mitoxantrone hydrochloride liposome injection orphan drug designation for treating peripheral T-cell lymphoma (PTCL).

In clinical practice, mitoxantrone is a widely used broad spectrum anti-tumor drug. Due to its severe adverse reactions of cardiotoxicity and myelosuppression, the clinical application is severely restricted. The liposome formulation of mitoxantrone has significant changes in pharmacokinetic, tissue distribution, pharmacological effects and toxicity. Its curative effects and safety increase significantly, comparing with the ordinary formulations.

Mitoxantrone hydrochloride liposome injection is developed solely by CSPC and there is no similar product reported beyond China. CSPC has independent intellectual property rights in this drug and it applies for 7 patents in China, 2 international patents and has obtained the warrant from EU and other 10 countries while still another 7 countries including USA and Japan are reviewing the application of this drug. The drug is on phase II clinical trial in China.

3.antibody conjugated drug DP303c

The antibody conjugated drug DP303c developed by CSPC obtained orphan drug designation awarded by FDA and is used for treating stomach cancer, including the gastroesophageal junction tumor.

DP303c is formed by a cytotoxin coupled with an antibody which is highly selective for HER2. It is a novel targeted drug for treating HER2-positive stomach cancer. DP303c showed promising anti-stomach cancer effects in the mouse model innoculated with human stomach cancer NCI-N87 cells and HER2 expressing SK-BR-3 cell line.

The antibody conjugated drug DP303c is developed solely by CSPC and has independent intellectual property rights. It applied a number of patents in many countries including the USA and China.

4.“ALMB-0166”Product Under Research

The product under research humanized gap junction protein 43 monoclonal antibody ALMB-0166,to treat SCI, obtained orphan drug designation awarded by FDA.

SCI is an emergency trauma, which may cause spinal contusion, partially or completely. It is also a common cause of children’s or adult’s permanent disability or death. ALMB-0166, by inhibiting cell hemichannel, has achieved effect in pre-clinical studies in preventing the formation of colloid scar after injury and promote functional recovery.

ALMB-0166 is an antibody drug developed by American branch Alamab Therapeutics, Inc. The branch concentrates on developing first-in-class antibody drugs.