BACKGROUND:
One of President Trump’s campaign promises was to shorten the approval process for new drugs, and that appears to be happening at a record pace.

AUGUST 30, 2018, BY TIM LAMMERS
The Food and Drug Administration said it approved 46 new drugs last year, the most since 1996, and also approved a record number of new generics, over a thousand. To do so, The F.D.A. streamlined its review and approval process, with the goal of getting generic drugs onto the market in eight to ten months, instead of the current average of four years. The idea is to let the influx of new drugs also lower the price for drugs as a whole.

“We're increasing competition. We're increasing the approval of new branded drugs, and bringing new therapies to market. So it's not going to change overnight,” said Department of Health and Human Services Secretary Alex Azar, “We are not driving for any kind of cheap gimmicks or quick solutions. We're doing things the right way.”

However, critics of the changes said the F.D.A. is simply lowering its standards, and putting consumers at risk for side effects.

“By allowing drugs to come to market ever faster we are exposing patients across the country to increased risks because we didn't learn about the harms that we could have learned had we done better and longer clinical trials before their approval," said Dr. Michael Carome, a drug and healthcare expert.

The F.D.A. said many of the newly approved drugs are designed to treat rare diseases, for which sufferers don’t have a lot of other options. Those people may be far more willing to risk side-effects.

https://fox61.com/2018/08/30/why-the-f-d...more-new-drugs/

TODAY:
Boston-Based Vertex's Drug for Cystic Fibrosis Patients Approved 5 Months Early
By Allison DeAngelis, Oct 22, 2019

Boston-based Vertex Pharmaceuticals Inc. has achieved one of the biggest feats to date for cystic fibrosis patients — a treatment that could benefit 90 percent of patients with the rare lung disease.

The FDA approved a combination of three drugs Monday called Trikafta, which targets a mutation in the F508del gene. That genetic error is found in approximately 90 percent of patients with the rare lung disease.

"If you think about it, we went from being able to treat no patients to being able to treat nearly all patients in seven years. That's never been done in the industry, to my knowledge," longtime CEO Jeff Leiden told the Business Journal.

There are currently around 30,000 people in the U.S. with the disease.

The drug is expected to be a blockbuster product for Vertex, which makes billions annually from its three existing products all for CF. Vertex has set a $311,000 annual price for Trikafta. The company's leading drug Orkambi, meanwhile, costs $273,000 each year.

Vertex (Nasdaq: VRTX) shares were up 4 percent before trading was halted around 3 p.m. Monday, then another 2 percent in after-market trading on Monday evening, increasing the value of the company by more than $2 billion on the news.

The approval's timing is unusual, coming five months before the FDA's deadline to make an approval decision on March 19, 2020.

“The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease,” Reshma Kewalramani, Vertex's current chief medical officer and incoming CEO, said in a statement.

Vertex began developing the triple combination therapy around four years ago.

Vertex is exploring potential gene editing, gene therapy or RNA-targeting treatments to address the remaining 10 percent of CF patients. Leiden estimates that it will take some 10 or 15 years to find a suitable drug.

In the meantime, Vertex hopes to launch Trikafta in Europe. The company has asked European regulators to approve the drug, but has yet to get any of its products off the starting block in England due to a longstanding insurance reimbursement dispute.

"I think our strategy has been working pretty well.... We will complete our discussions with (existing products) Orkambi and Symdecko before discussing Trikafta," Leiden said. When asked if the company planned to use Trikafta as incentive for British regulators to approve reimbursement on the company's pre-existing products, Leiden said "I don't think it's about leverage. It's about them understanding the values of our medicines."

https://www.bizjournals.com/boston/news/...t_news_headline
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FUNNY: They completely neglect to give any credit to Orange Man. TM