Drug for Rare Genetic Disease Will get Food and drug administration Approval


Pathway to Approval: The Story of a Rare Disease Drug

Video taken from the channel: Takeda


FDA Approves Gene Therapy for Leber’s Congenital Amaurosis

Video taken from the channel: Penn Medicine


A New FDA-Approved Gene Therapy Helps Treat a Rare Childhood Cancer

Video taken from the channel: U.S. Food and Drug Administration


Why FDA’s approval of genetic therapy is such a big deal

Video taken from the channel: CBS Evening News


Red Flag Over Biogen’s Expensive Drug For Rare Genetic Disease | Power Lunch | CNBC

Video taken from the channel: CNBC


FDA Approves World’s Most Expensive Drug To Treat Rare Disease | NBC Nightly News

Video taken from the channel: NBC News


FDA approves Novartis gene therapy for rare genetic disorder

Video taken from the channel: CNBC Television

The U.S. Food and Drug Administration today approved Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults with Fabry.

ByLinda Carroll and Lauren Dunn The Food and Drug Administration has approved a drug to treat a rare and fatal genetic disease in children, a life-saving therapy that will cost $2.1 million. The. The FDA has approved a new indication for drug to treat seizures associated with tuberous sclerosis complex.

TSC is a rare genetic disease that causes non-cancerous (benign) tumors to grow in. FDA Approves New Indication for Drug Containing an Active Ingredient Derived from Cannabis to Treat Seizures in Rare Genetic Disease News provided by U.S. Food and Drug Administration. FDA approves new treatment for rare hereditary disease [8/23/2018] The U.S.

Food and Drug Administration today approved Takhzyro (lanadelumab), the first monoclonal antibody approved in. FDA Approved Drugs The following database contains a listing of drugs approved by the Food and Drug Administration (FDA) for sale in the United States. Drug information typically includes the drug name, approval status, indication of use, and clinical trial results. The U.S.

Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular. May 24, 2018 The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as phenylketonuria (PKU).

The Orphan Drug Act is a law passed by Congress in 1983 that incentivizes the development of drugs to treat rare diseases. Companies and other drug developers can request orphan drug designation. GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases.

The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product.

List of related literature:

Currently, the FDA is exploring the role that it might play in regulating genetic testing, which has put a hold on direct-to-consumer genomic testing for disease risk assessment.

“Swaiman's Pediatric Neurology E-Book: Principles and Practice” by Kenneth F. Swaiman, Stephen Ashwal, Donna M Ferriero, Nina F Schor, Richard S Finkel, Andrea L Gropman, Phillip L Pearl, Michael Shevell
from Swaiman’s Pediatric Neurology E-Book: Principles and Practice
by Kenneth F. Swaiman, Stephen Ashwal, et. al.
Elsevier Health Sciences, 2017

It may even be possible to convince the FDA to reconsider a rejected drug if test results are reconfigured to display the benefits among patients shown to be appropriate through genetic screening.

“The Biotech Investor's Bible” by George Wolff
from The Biotech Investor’s Bible
by George Wolff
Wiley, 2001

The first drug approved in 2010 was trastuzumab, a monoclonal antibody against Her2, which is over-expressed in approximately 20% of gastric cancers.

“Abeloff's Clinical Oncology E-Book” by John E. Niederhuber, James O. Armitage, James H Doroshow, Michael B. Kastan, Joel E. Tepper
from Abeloff’s Clinical Oncology E-Book
by John E. Niederhuber, James O. Armitage, et. al.
Elsevier Health Sciences, 2019

Of note, this was the first time that the FDA recommended genetic screening for a major drug.

“Lehne’s Pharmacotherapeutics for Advanced Practice Nurses and Physician Assistants E-Book” by Laura Rosenthal, Jacqueline Burchum
from Lehne’s Pharmacotherapeutics for Advanced Practice Nurses and Physician Assistants E-Book
by Laura Rosenthal, Jacqueline Burchum
Elsevier Health Sciences, 2017

The U.S. Food and Drug Administration (FDA) has approved several genetic tests for cytochrome 2D6 and 2C19 variants as part of its efforts to improve patient safety in the treatment of human immunodeficiency virus/acquired immunodeficiency syndrome (HIV/AIDS).

“Murray and Nadel's Textbook of Respiratory Medicine E-Book: 2-Volume Set” by Robert J. Mason, V.Courtney Broaddus, Thomas R Martin, Talmadge E King, Dean Schraufnagel, John F. Murray, Jay A. Nadel
from Murray and Nadel’s Textbook of Respiratory Medicine E-Book: 2-Volume Set
by Robert J. Mason, V.Courtney Broaddus, et. al.
Elsevier Health Sciences, 2010

Oktay Kutluk

Kutluk Oktay, MD, FACOG is one of the world's foremost experts in fertility preservation as well as ovarian stimulation and in vitro fertilization for infertility treatments. He developed and performed the world's first ovarian transplantation procedures as well as pioneered new ovarian stimulation protocols for embryo and oocyte freezing for breast and endometrial cancer patients.

Mail: [email protected]
Telephone: +1 (877) 492-3666

Biography: https://medicine.yale.edu/profile/kutluk_oktay/
Bibliography: oktay_bibliography

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  • Most new drugs come from U.S.A. https://xconomy.com/seattle/2014/09/02/which-countries-excel-in-creating-new-drugs-its-complicated/
    Only 1 in 5000 new drugs make it to market and then it takes 12 years: https://www.medicinenet.com/script/main/art.asp?articlekey=9877

    You have to pay for all that inorder to reap the benefits of all that. If you don’t pay for that you get none of it. Simple as that. Durgs take money to make. No money means no drugs. This things don’t grow on trees. The genetic stuff is especially expensive to make because it takes very complex machines to make.
    I have sympathy for genetic diseases. No one asked for it. Certinaly not the baby. But if you want the drugs for your cholesterol, high blood pressure, liver disease, and kidney problems let me suggest to you that you put down that big mac, return that 6-pack to the 7-11, and stop wining and dining out.
    Especially stop wining.

  • Big pharma RIP off…it should be outlawed!!
    It’s ironic how the father said he would do whatever possible for his son…
    Well…he didnt have to pay the 2.1 million..it was charity!! I am sure he wouldn’t say the same thing if the money was coming out of his pocket. Go figure!

  • The problem is the company may spend hundreds of millions to bring a new drug to market that is only rarely going to be used, and it has to recoup that money plus make a reasonable profit before there patent runs out and it is cloned.

  • The real problem with drug price is the artificial separation created by programs like Medicaire Part D and market forces. All it takes, is bribe the right politician and you product would be covered by the government! In the case of pharmaceuticals, the politicians were the Republicans and the Bush Administration. It is not just limited to Democratic politicians. If you limit the import of cars, then American cars would cost over a million too!

  • Yea but he wouldent be saying that when you have nothen left after selling everything and leaving on the streets becouse we let then big wigs but a tax on everything including soon tax on air.

  • In Portugal all portuguese people stay togheter and everybody is helping a baby with this disease donating to reach the 2 million. Search in facebook “Matilde uma bébé especial”

  • New Zealand has a tax payer funded public health care system with supplementary private sector.
    The budget for a liver transplant for example, is around 100 thousand dollars, not the 3 to 5 million it costs in the US. New Zealand also has a government drug buying agency that negotiates with the pharmaceutical companies for the good of the entire population, not just the rich. Let’s hope New Zealand can hold on to its human decency.

  • As a biomedical scientist, I am fascinated by news in the healthcare/pharma sector. Which is why I have recently made a video on this subject. Check it out: https://youtu.be/594kZ_cW83c

  • Bogus.
    Any so-called miracle drugs, are possible because of huge scientific/technology breakthrough funded by public money around the globe. The drug makers only finished the last stretch, which is tiny compared with has been achieved before them. If they don’t do it, somebody else will, just a matter of time.

  • So because it does such an amazing thing it shouldn’t be affordable?? U know people want it, u know people really need it, and so u know people will definitely try and or pay for it. Disgusting

  • A lot of money in research and time is spent on very rare diseases

    isn’t that an interesting concept

    Who’s paying for all that research and why aren’t the pharmaceuticals paying back the American hard working taxpayers for all that free money we gave them for research

    compound with interests

    and long term viability for the American people of this country that supported, a nation of peacefulness, so you corporate greed can take advantage of the whole world!?

    not no more you corporations

    you like safety go find another country to operate in

    Don’t be coming over here and mouchon off U.S. no more…

  • Nearly one billion turnover in the first year if all children are treated. 5 years write off would mean that there were 5 billion in costs to design this medicine???? Really?

  • FAKE NEWS NBC….still supporting the criminals @ Big Pharma

    A marriage made in heaven to screw the American people over.

    Trump 2020

  • So good to see new medications coming out to saves lives…. Soon after 10 to 20 years it will affordable to the poor.. God bless America

  • No price on human life?….unless you’re a snake providing medication in America…..that price then is 2.1 million
    ……meanwhile after they win their bread and butter it’s only $500 in 3rd world countries. Am I wrong? Nope, I’m not.

  • This drug has major effects so they see this as an opportunity and try to groom peoples head by saying you should pay this price for the drug. P.S save lives with morals not businesses.

  • All these big Pharma Executives ought to be lined up and shot. I can’t remember which one but his salary is 100 million. Nobody is worth that much at work

  • very very expensive that s for the most richeast personality……how about poor people?….. that s a big business for da money maker and true hero what!!!!!!!!!!

  • I’m from India more people are suffurring here with muscular distrophy ND atrophy pateints need to alive please discuss to all countries of the governments��������������������

  • It’s great to see U.S. taxpayers paying extremely redickuless prices for medications, that the taxpayers gave the GRANTS to develop.
    Makes sense to me