Orphan Medicine Weekly | Innovative immune-activated cells, 8 therapies, such as recombination proteins...

Last week the FDA awarded eight orphan drug qualifications, including Amgen's recombinant protein drug romiplostim, Immunicum's immunoactivated cell therapy ilixadencel, and Vifor Pharma's Relypsa-developed sickle cell anemia oral small molecule therapy VIT-2763.
today's article the Drug Ming Kant content team will do an inventory for you.
Drugs: Talabostat Research and Development: BioXcel Therapeutics Treatment: Soft Tissue Sarcoma (STS) Introduction: Soft tissue sarcoma (STS) is a group of malignant tumors derived from connective tissue such as fat, blood vessels, and lymphatic tubes, including neuroblastoma originating in the outer neuroblast.
the disease can occur in any age group, can occur in almost any part of the body, its pathogenesis and pathogenesis is still unclear.
non-differentiated sarcoma often has a higher regional lymph node metastasis rate, and the prognosis is very poor once the metastasis occurs.
the lungs in distant transfer areas, followed by the bone, liver and brain.
Talabostat is a small molecule with anti-tumor and hematologic functions that inhibits the activity of dispeptide-based peptide enzymes (such as fibroblast-activated proteins) by cutting N-side Xaa-Pro or Xaa-Ala residues, thereby stimulating cytokine and polymerization factor production and specific T-cell immunoactive activity.
the drug can also stimulate the production of set-up stimuli, such as granulocyte-set stimulating factors (G-CSF), thus playing a hemato-forming role.
: Suramin Research and Development: Pax Medica Treatment: Human Trypanosomiasis (HAT) Introduction: African Human Trypanosomiasis (HAT) Introduction: African human trypanosomiasis, also known as sleeping sickness, is a parasitic disease.
the disease is caused by a primary parasitic infection of trypanosomiasis.
these parasites are transmitted to humans through the bites of tsetse flies.
has been used as standard treatment for early East African sleeping sickness for more than 100 years by blocking radon signaling pathflights to stop parasites from proliferating.
PaxMedica will use its patient data for the treatment of early sleeping sickness in East Africa to develop the intravenous preparation PAX-101, which is in Phase 3 clinical studies for the treatment of human trypanosomiasis in Africa, according to its website.
Drugs: Ilixadencel Research and Development: Immunicum Treatment of Diseases: Soft Tissue Sarcoma (STS) Introduction: Ilixadencel is a ready-to-use immunoactivated cell used to treat soft tissue sarcoma (STS) including malignant gastrointestinal mesothelioma (GIST).
Lixadencel's active ingredient is derived from activated isotrophy dexterous cells from healthy blood donors that, when injected into a patient's tumor, induce an inflammatory response at the tumor site, which in turn induces tumor-specific activation of the patient's cytotoxic T-cells.
in previous studies, ilixadencel combined standard therapeutic drugs and immuno-checkpoint inhibitors to treat the safety and tolerance of multiple solid tumors, and Immunicum is currently advancing its late-stage clinical development.
: VIT-2763 Research and Development Enterprise: Relypsa (Vifor Pharma Subsidiary) Treatment of Disease: Sickle Cell Anemia (Sickle Cell Disease, SCD) Introduction: Sickle Cell Anemia (Vifor Pharma Subsidiary) SCD) is a hereditary hemoglobin (Hb) disease caused by the replacement of glutamate in the 6th bit of the β-peptide chain with proline, which causes hemoglobin abnormalities to become known as sickle-like red blood cells.
SCD belongs to the normal chromosomal explicit inheritance, the incidence rate is about 8 cases per 100,000 people, clinically mainly manifested as chronic hemolytic anemia, chronic local ischemia leads to organ tissue damage, infection and recurrence of pain risk.
VIT-2763 is an oral ferroportin inhibitor with the potential to treat erythrocyte production and iron overload diseases.
transport proteins play a key role in regulating iron intake and distribution in the body.
VIT-2763 binds and blocks ferrotransering proteins to prevent excessive iron release into the bloodstream.
June 2019, the FDA and the European Medicines Agency (EMA) have granted VIT-2763 eligibility for orphan drugs for the treatment of β-thalassemia.
Drugs: Romiplostim Research and Development Enterprise: Amgen Treatment of Diseases: Thrombocytopenia Caused by Chemotherapy Introduction: Rombocytopenia Introduction: Rombocytopenia is a recombinant protein that belongs to plateocyte-producing (TPO) subjectors that stimulate in-cell transcription by binding to TPO subjects, resulting in an increase in plateplate production.
the drug, produced in E. coli by recombinant DNA technology, can simulate the body's natural plateplate-producing effects, thereby increasing plateboard counts.
When combined with a TPO subject, it promotes the growth of bone marrow giant nucleocytes (colony-forming unit-megakaryocyte, CFU-Meg) and leads to an increase in plate plate production through JAK2 and STAT5 kinase pathways.
drugs: saroglitazar magnesium Research and Development Enterprise: Zydus Discovery DMCC Treatment: Primary bile bile cholangitis Introduction: primary bile bile dulineitis a.k.a. primary bile cirrhosis, is an autoimmune disease that occurs in the liver.
patients' bile tubes that deliver bile acid outwards in the liver are subjected to an abnormal attack on their own immune system, causing substances such as bile acid to accumulate in the liver, causing liver damage, which in turn can lead to liver fibrosis and cirrhosis.
Saroglitazar magnesium is a new type of peroxidase proliferative activator (PPAR) astracite, which has the function of regulating the activity of PPAR alpha and PPAR gamma.
PPAR alpha/γ is an important transcription factor in regulating the body's metabolism and plays an important role in fat metabolism and insulin resistance.
previous non-clinical data show that saroglitazar magnesium's unique dual mechanism of action is more effective in treating diseases than pure PPAR alpha or PPAR agonists.
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
resources: novartis Gene Therapies. Retrieved Jan 22, 2021,from Amgen. Retrieved Jan 22, 2021,from Aclaris. Retrieved Jan 22, 2021, from Yasheng Pharmaceuticals won the 9th FDA Orphan Drug Qualification, the largest in China. Retrieved Jan 22, 2021,from Q BioMed. Retrieved Jan 22, 2021,from Smart Immune. Retrieved Jan 22, 2021,from Ayuvis. Retrieved Jan 22, 2021, from