Support seen for U.S. plan to speed nods for ‘breakthrough’ drugs

Posted under Blog, Cancer Drugs, Companies, Diagnostics, FDA, Funding, HIV, Janet Woodcock, Kalydeco, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Regulatory, Startups, Universities, Videos, Xalkori, Zelboraf by rmcbride

Drug companies have clamored for the FDA to provide a more efficient pathway to gain product approvals, and this year the agency and U.S. lawmakers appear to be supportive of a plan that would help pharma groups race to the market with breakthrough drugs.

As Reuters reports today, the plan is expected to become law this year, formalizing a regulatory pathway that allows the FDA to let drugmakers conduct speedier trials with fewer patients prior to approval when their treatments provide major benefits for patients. The system would be akin to the agency's accelerated approach to ushering life-sustaining HIV treatments onto the market.

U.S. regulators have shown a willingness to take urgent action on reviews of breakthrough drugs. For instance, the agency stamped an approval of Vertex's ($VRTX) cystic fibrosis therapy Kalydeco after a quick three-month priority review rather than the standard 10-month review period. And the agency has acted quickly on applications for string of cancer meds such as Pfizer's ($PFE) lung cancer drug Xalkori and Roche's ($RHHBY) Zelboraf for skin cancer that showed impressive results in the clinic, Reuters reported.

Such approvals helped the industry see an uptick in market nods for new drugs, yet there are still significant barriers in place that keep development cycles even for powerful new meds long and expensive. Dr. Janet Woodcock, the FDA's top drug regulator, has been a vocal advocate for giving the agency greater latitude to approve breakthroughs.

"What happens when you have a breakthrough drug that shows an effect that's never been seen before?" Woodcock said in March, as quoted by Reuters.

- check out Reuters' article

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UPDATED: Vertex sizzles with upbeat PhII data on combo therapy for cystic fibrosis

Posted under Blog, Clinical Trials, Companies, cystic fibrosis, Diagnostics, Funding, Kalydeco, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos, VX-809 by rmcbride

Vertex Pharmaceuticals ($VRTX) has nailed down some promising mid-stage data for a combo treatment against the genetic lung disorder cystic fibrosis. An interim analysis of the Phase II study showed that patients on the company's drug Kalydeco and an experimental treatment had significantly improved lung function compared with those on placebo, Vertex announced.

The data indicate that Vertex might have a remedy for treating a large number of the 70,000 people worldwide with CF, and the company's share price had jumped nearly 46% to $54.69 as of 12:16 pm ET today. 

There was a 5% improvement in lung function in 46% of patients on the recently approved Kalydeco and VX-809, and 30% of patients on the combo saw at least a 10% boost in lung function, according to the company. A 4%-6% improvement in lung function in the study would be a "home run," an analyst from RBC Capital Markets wrote last month, according to Bloomberg's report on the study. Yet Forbes' pharma editor Matthew Herper cautions that the study isn't complete and the combo hasn't met everyone's expectations.

Once the company wraps the Phase II study and talks to regulators, Vertex aims to race ahead with a pivotal study of the Kalydeco-VX-809 combo for treating the root cause of the genetic disease in patients with two copies of the F508del mutation. Almost half of all patients with the disease, which causes thick and sticky mucus to build up in the lungs, have two copies of the mutation, according to the company. Kalydeco, the first drug approved to combat the underlying cause of CF, has approval for treating patients with a mutation that signifies about 4% of patients with the disease, Bloomberg reported.

"People with two copies of the F508del mutation have one of the most severe forms of cystic fibrosis. In these patients, the combination of VX-809 and Kalydeco led to significant improvements in lung function that exceeded our expectations," Dr. Chris Wright, Vertex's senior vice president, global medicines development and medical affairs, stated. "We look forward to beginning discussions with regulatory agencies later this year when we have full data from the study, with the goal of moving forward with a pivotal study as quickly as possible."

The FDA has shown that the agency will take quick action on drugs that prove effective against life-threatening diseases such as CF, which leads to premature death. As Bloomberg noted, the agency stamped an approval on Kalydeco after a three-month review.

- here's the release
- check out Bloomberg's article
- see Herper's piece
- and TheStreet's coverage

Related Articles:
Vertex nabs expedited FDA OK on breakthrough CF drug
Vertex backs up pricey new CF drug with co-pay help
One father's heartbreak inspired a $250M quest for a cure

Editor's note: Updated stock price at 12:20 pm ET on 5/7/12.

One father’s heartbreak inspired a $250M quest for a cure

Posted under Blog, Companies, cystic fibrosis, Diagnostics, Funding, Kalydeco, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by John Carroll Every new drug ever approved involved years of work by dedicated teams of investigators. In the case of the new cystic fibrosis drug Kalydeco from Vertex ($VRTX), the story also includes Boston businessman Joe O'Donnell, who helped raise much of the money the Cystic Fibrosis Foundation used to back the development of the treatment. The Boston Globe today tells the touching story of O'Donnell's passionate commitment to getting the treatment pushed to an FDA approval. The groundbreaking treatment heralds the approach of a new generation of therapies intended to help treat the full range of CF patients. And that's possible because O'Donnell devoted much of the past 30 years to raising more than $250 million for the foundation following the death of his young son Joey, a victim of CF. "We would not be where we are, had he not been there,'' foundation CEO Robert Beall tells the Globe. "He's authentic. He's a man of his word. He's not going to ask anybody for anything that he doesn't believe in.'' It all hit home when one of the parents of a young CF victim told O'Donnell how much the new treatment had helped. "That was the moment for me,'' O'Donnell said. "I talked to Gerry (Cheevers) and, my God, the dam broke. You get emotional. It's a long slog, and I think of Joey. It's one of the reasons he was put on this Earth.'' - here's the Globe article Related Article: UPDATED: Vertex nabs expedited FDA OK on breakthrough CF drug

Kalydeco ranks among 12 priciest drugs in U.S.

Posted under Blog, Companies, Diagnostics, drug prices, Funding, Kalydeco, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by John Carroll

Last week, Vertex Pharmaceuticals ($VRTX) got FDA approval for Kalydeco, the first cystic fibrosis drug to address the disease's cause, rather than just its symptoms. The price tag: $294,000. But as observers pointed out at the time, that's not as expensive as it gets. Today's drugs for debilitating and rare diseases command 6 figures almost as a matter of course. Medical Billing & Coding's blog reminds us just how much--and shows where Kalydeco ranks. Story