The Many Perspectives of the FDA Approval of ORKAMBI

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by Stephen Shannon |

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On Thursday, July 2, the Food and Drug Administration made the announcement that Orkambi had been approved for people with cystic fibrosis who are age twelve and older with two copies of the F508del mutation. The announcement was followed by a swirling of excitement through the CF community and media outlets praising the decision and the benefits it is sure to bring to nearly half of the current CF population.

Each month I provide a series of readings that highlight the news stories relevant to the progress of CF drug development and healthcare. For this month I have provided four articles that tell the story of Orkambi’s release through the many perspectives of the news, government, patients and CF Foundation. I would encourage you to take time to follow the links provided to read more of the articles that suite your specific interests.

FDA approves new treatment for cystic fibrosis

Source: U.S. Food and Drug Administration

In a press release issued on Thursday, July 2, the FDA announced the approval of Vertex Pharmaceutical’s breakthrough designated drug, Orkambi. The release serves as an electronic monument to the funding and work that was poured into the drug’s development by researchers, those working for the CF Foundation, patients and families.

The FDA, in the release, takes note of the accomplishment achieved in the CF community. “Orkambi received FDA’s breakthrough therapy designation because the sponsor demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies.” They go onto explain, “the FDA also reviewed Orkambi under the priority review program. A priority review is conducted over six months, or less, instead of the standard 10 months, and is employed for drugs that may offer significant improvement in safety or effectiveness in treatment over available therapy in a serious disease or condition.”

The release continues, detailing the drug’s indications and the CF population it is targeted to treat. It is well worth the read to get a general overview on the approval and the benefits for patients.

Will I live long enough for someone to give me his seat on the subway?

Source: Washington Post

This article shifts from the bureaucratic announcement of the drug to a story provided by one patient who has already begun to feel relief from Orkambi. The article shares a candid account of living with CF during a time when new medications may provide a future and old age.

Steven Cooney, the writer of the recent article in the Washington Post, tells his story living with the disease and his changing thoughts for what his future may look like. Cooney was one of the few patients who was enrolled to take Orkambi during clinical trials and has already begun to benefit from its use. “In recent months, I’ve spent days feeling better than I have in years. Other days reminded me that this is still cystic fibrosis, and we have a long way to go.”

Letter to the Community on Orkambi Approval

Source: Cystic Fibrosis Foundation

The third article arcs from the story of a patient to a letter written by Robert J. Beall, Ph.D, president and CEO of the CF Foundation. The CF Foundation under the leadership of Beall has been a pioneer in providing funding to accelerate the development of drugs for use in those with CF. The letter by Beall is written to everyone who has helped push for the cause of CF through research, advocacy, giving and volunteering.

“Today’s announcement gives us much to be proud of, along with tremendous momentum as we move forward urgently in our efforts to find powerful new therapies for all people with CF.”

The letter acknowledges that the end has not come but there is much left to be accomplished for everyone with CF but there is tremendous promise through the work that is being done by the Foundation. The foundation commented that, “Orkambi is the latest example of the CF Foundation’s venture philanthropy model, through which it has invested hundreds of millions of dollars to help develop CF drugs and therapies. This approach has led to tremendous advances in life expectancy for people with CF, which has doubled in the past 30 years. Nearly every CF drug available today was made possible because of Foundation support.”

Are risks worth the rewards when nonprofits act like venture capitalists?

Source: Washington Post

The final article shifts from the experiences of those who took part in the approval of Orkambi to what the future holds with the current venture philanthropy model used by the CF Foundation. Orkambi as well as Kayldeco before it was brought to market in part from the funding of the CF Foundation Therapeutics which is an arm of the CF Foundation which provides grants for CF related research. In recent years the foundation has drawn critics by extending their giving beyond academic labs to pharmaceutical companies in a push to get drugs for CF patients into market more quickly.

“Frustrated that no game-changing treatments were in sight, the group’s leaders in 1999 placed what many considered a risky bet, deciding to invest millions of dollars in a small California biotech firm. Robert J. Beall, the foundation’s president, believed that putting money into drug companies directly, rather than merely making grants to academic investigators, might persuade the industry to focus on the disease and turn research into real-world treatments.”

Detractors of the venture philanthropy model argue that, “patient groups risk harming their reputations and their bank accounts by forming partnerships with the drug industry. Some experts worry that the relationships create inherent conflicts of interest and that nonprofits could allow financial motives to undermine their primary mission of putting patients first.”

In addition to the coverage noted above, Cystic Fibrosis News Today has offered leading, comprehensive coverage of Orkambi’s testing and eventual approval. To read previous news coverage of Orkambi and follow continuing coverage of the therapy’s impact on the CF patient population, follow the Cystic Fibrosis News Today news tag page for Orkambi.

Regardless of the arguments, it is undeniable that without the funding of the CF Foundation the development of both Kayldeco and Orkambi would not have been possible, leaving no room for the current celebration within the CF community.