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May 07, 2019
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A new drug co-developed by Roche and Ionis Pharmaceuticals can be a potential candidate to treat Huntington’s disease. RG6042 which is an antisense oligonucleotide is able to successfully lower down the production of a toxic protein that results in dysfunction of neurons. The successful Phase 1/2a trials have boosted the hopes of Roche to take the drug directly in Phase III trials.
Once rejected by the U.S. FDA, Pfizer’s tafamidis recently was able to win the FDA nod to treat transthyretin amyloid cardiomyopathy, a rare and fatal heart disease. The oral drug will be sold under the brand name Vyndaqel is set to bear a price tag worth USD 225,000. The dug has been set a price which is much lower than that of Tegsedi a collaborated product of Akcea Therapeutics and Ionis Pharmaceutical and Onpattro of Alnylam Pharmaceutical.
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Syapse, a precision medicine software developer has announced to collaborate with Amgen to advance in cancer drug development. With the help of Syapse’s health network, Amgen aims to identify the patients suitable for clinical trials and select the potential drug candidates. Both the companies have decided to work towards the developing of real-world evidence standards to support the acceleration of therapies to market.
Jacobus Pharmaceutical recently was able to win FDA’s approval for its drug Ruzurgi for the treatment of a rare autoimmune disorder Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. The drug is the first ever approved treatment, especially for pediatric patients. Ruzurgi received an ORPHAN designation along with Priority review and Fast Track designations.
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