And PRIME still hasn’t changed their 500k cost prediction(funny or serious?)
https://www.primetherapeutics.com/en/services-solutions/connect/2017/congenital_plasminogen_deficiency.html PRIME estimates Ryplazim will cost $500,000 annually
Drug watch list: congenital plasminogen deficiency
December 21, 2017
Staying ahead so our clients don’t fall behind
At Prime Therapeutics (Prime), we’ve positioned ourselves ahead of drug trends to best prepare for new, high-cost drugs entering the market. Our drug watch list is managed as a cross-functional effort with our clinical, formulary, Health Outcomes, and specialty teams.
New to the watch list:
Drug: RyplazimTM
Manufacturer: Prometic Life Sciences
Condition: congenital plasminogen deficiency
Condition overview:
Plasminogen is a protein synthesized in the liver that is vital in wound healing. Congenital plasminogen deficiency is a rare genetic disorder caused by an alteration to the plasminogen gene. These alterations can result in inflamed growths on the mucous membranes, such as the eyelids and the inside of the mouth. This can cause scarring of the cornea, blood clots and closing of the windpipe, especially in children.1
Current treatments:
Due to the rarity of the disease, there are no standard treatment procedures. Several therapies have been reported in single-case or small cohort studies, but have not been proven. Growth-removal surgery is also common, but growths usually reoccur.2
Ryplazim overview:
Prometic Life Sciences, Inc. (Prometic) has stated that they have created a process to extract, isolate and purify high-value proteins with a yield and efficiency superior to what is currently available. Prometic also stated that, in addition to being safe, well tolerated and without any drug-related, serious adverse events, Ryplazim achieved a 100% success rate in its Phase 2/3 trial.3
The trial was held over a total of 48 weeks and included 10 patients. Ryplazim reached its targeted increase in the blood plasma concentration level of plasminogen.4 In addition, the trial showed that Ryplazim treatment resolved all lesions in patients treated.5
PDUFA Date:
4/18/2018
Costs:
Prime estimates Ryplazim will cost $500,000 annually.
The drug pipeline is full of new, groundbreaking specialty drugs that may help members live better and feel well. Our drug watch list is just one of our clinical strategies designed to keep you ahead of drug trends — because it’s easier to manage change when you see it coming.
Contact your Prime representative for more information, or for the full drug watch list.
- 1. https://rarediseases.org/rage-diseases/congenital-plasminogen-deficiency
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- 2. https://www.prometic.com/prometic-announces-positive-long-term-clinical-data-on-ryplazymtm-in-plasminogen-congenital-deficiency-and-provides-regulatory-update/
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- 3. https://rarediseases.org/rage-diseases/congenital-plasminogen-deficiency
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- 4. https://prometic.com/therapeutics/plasminogen/
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- 5. https://www.epgonline.org/global/news/fda-accepts-application-for-ryplazim--plasminogen-replacement-therapy--in-plasminogen-deficiency---prometic-life-sciences-.html
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