What is arimoclomol used for?
Arimoclomol is an experimental drug compound developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California. The orally administered drug is intended to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, a neurodegenerative disease with no effective treatment.
Will arimoclomol be approved?
The U.S. Food and Drug Administration (FDA) has denied the approval of arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C (NPC). NPC is a disabling neurogenetic disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood.
Who makes arimoclomol?
CytRx Corporation
Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) is an experimental drug developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California.
Is masitinib FDA approved?
04/10/2021 – AB Science today announced that its clinical development program of masitinib in adult patients with mast cell activation syndrome (MCAS) has been approved by the U.S. Food and Drug Administration (FDA)
What are the results of the research on arimoclomol for inclusion body myositis?
Developed by the Danish laboratory Orphazyme, arimoclomol enhances the production of heat shock proteins. It had obtained encouraging results in inclusion body myositis in preclinical studies, but its efficacy was not confirmed in a small clinical trial (24 participants) which evaluated it at a dose of 300 mg / day.
What is the difference between IBM and ALS?
“You have IBM [inclusion body myositis].” Like ALS, IBM is a progressive neuromuscular disorder, but unlike ALS, it’s not fatal. Further tests, including a muscle biopsy, confirmed the diagnosis. “I’ve had ups and downs throughout my life,” Frampton says.
Will FDA approval Orphazyme?
The FDA has rejected the therapy and asked Orphazyme to provide additional data on the benefits and risks of the heat shock protein amplifier, leading the company to cut costs as the next steps are considered.
Did FDA approve Orphazyme?
In the announcement of the FDA rejection, Orphazyme CEO Christophe Bourdon said the company will need to “reduce our costs substantially and freeze all company efforts not related to clinical and regulatory activities to support approval for [Neimann-Pick disease type C].”
What is Adrabetadex?
The drug promotes transport of the cholesterol that would normally accumulate in the lysosomes of cells that have the NPC1 or NPC2 mutation. The company is additionally developing a device to assist in administration of the product.
What is Orphazyme arimoclomol?
Arimoclomol is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins and improve the function of lysosomes. Arimoclomol is administered orally, and has now been studied in 10 Phase 1, four Phase 2, and three pivotal Phase 2/3 trials.
What company makes masitinib?
AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases.
Is masitinib available?
The drug is available to treat cancer in veterinary medicine under the names Masivet or Kinavet. For all trials of masitinib in Alzheimer’s, see clinicaltrials.gov.