NorthWest Biotherapeutics Inc (NWBO)

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FDA Approves 5 New Costly Drugs Well Ahead of PDUFA Dates
Posted 26 November 2019 | By Zachary Brennan 

https://www.raps.org/news-and-articles/news-articles/2019/11/fda-approves-5-new-costly-drugs-well-ahead-of-acti

Since 21 October, the US Food and Drug Administration (FDA) has been on a tear in approving five new drugs (all with list prices of more than $100,000 per year) months ahead of when they were expected to be approved.

For instance, FDA signed off on Vertex Pharmaceuticals’ Trikafta (elexacaftor/ivacaftor/tezacaftor), a new treatment for those with the most common cystic fibrosis mutation, after only three months of review and well ahead of its 19 March 2020 user fee action date.

On 14 November, more than three months ahead of its 27 February 2020 action date, FDA granted accelerated approval to BeiGene’s Brukinsa (zanubrutinib) for the treatment of patients with mantle cell lymphoma who have received at least one prior therapy.

One day later, Novartis’ Adakveo (crizanlizumab-tmca) won approval for its sickle cell disease treatment two months ahead of its PDUFA date in mid-January 2020. And yesterday, FDA granted an accelerated approval to another sickle cell drug, Global Blood Therapeutics’ Oxbryta (voxelotor), three months ahead of its PDUFA date.

Alnylam Pharmaceuticals’ Givlaari (givosiran), meanwhile, had a PDUFA date of 4 February 2020, but won approval on 20 November. But other recent approvals, like SK Life Sciences’ Xcopri (cenobamate tablets) to treat partial-onset seizures in adults, and Shionogi’s complicated urinary tract infection drug Fetroja (cefiderocol), won approvals near their PDUFA dates.

The string of quick approvals may provide more ammunition for those who criticize the agency for moving too quickly. An article in JAMA Internal Medicine last summer found that few cancer drugs approved via the accelerated approval pathway improved survival in confirmatory trials.

However, viewers of the recent Senate committee hearing considering a new FDA commissioner have seen there are still senators who believe FDA is not moving quickly enough with some approvals.

As the proportion of new drugs receiving expedited approvals in recent years has been increasing, so has the number of approvals for rare diseases.

Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, explained recently that the agency is working on its own analyses to provide “a more robust response” to these critiques of its approval standards.

She also explained how the high number of approvals in recent years for rare diseases may be influencing this perception of a lower bar, especially as more treatments are approved on the basis of a single-arm study or with an external control group. In addition, she pointed to the “astoundingly” high launch prices for some of these rare disease treatments that may also be part of the reason for the pushback.

Indeed, before discounts, Trikafta will cost $311,503 annually, Brukinsa will cost $12,935 for a 30-day supply, Adakveo will cost between $7,000 and $9,500 per month ($84,000 to $114,000 per year), Oxbryta will cost $125,000 per year and Givlaari will cost $575,000 per year. 



FDA issues speedy approval of innovative sickle cell drug
By Meredith Wadman Nov. 26, 2019

https://www.sciencemag.org/news/2019/11/update-fda-issues-speedy-approval-innovative-sickle-cell-drug

The U.S. Food and Drug Administration (FDA) approved the sickle cell disease medication Voxelotor for market on 25 November, fully 3 months ahead of a statutory deadline for agency action. “Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder,” acting FDA Commissioner Brett P. Giroir said in a statement.


FDA hastily approves Daiichi Sankyo/AZ’s cancer drug, weeks after US filing
December 23, 2019

https://pharmaphorum.com/news/fda-hastily-approves-daiichi-sankyo-azs-cancer-drug/

The FDA has approved an advanced breast cancer drug from AstraZeneca and Daiichi Sankyo in super-quick time.

The regulator has okayed the trastuzumab deruxtecan a few weeks after receiving a file making the case for it in metastatic HER2-positive disease in patients who have received two or more anti-HER2 based regimens. Branded as Enhertu (fam-trastuzumab-deruxtecaen-nxki), the drug consists of the antibody used in Roche’s Herceptin, linked to a topoisomerase inhibitor that is toxic to cancer cells. It therefore works by latching on to the cancer cells and delivering a payload to kill them, while ignoring healthy cells, and is designed for patients who have failed to respond to Roche’s HER2-targeting cancer drugs Herceptin, Perjeta, and Kadcyla.

The speed of approval shows just how keen the FDA is to new and effective cancer drugs – Daiichi only filed it in October. Faster Priority Reviews allow for approval within six months, but the FDA’s super-fast decision paves the way for a launch in the coming weeks. This has also been one of the fastest developments of a biologic drug, as Daiichi only began clinical trials four years ago.

AstraZeneca earlier this year asked investors for an extra $3.5 billion to finance a development and collaboration deal with Daiichi over the drug, paying $1.35 billion up front and up to $5.55 billion upon achievement of regulatory and other milestones. Under the agreement the two companies will split the development and marketing costs as well as global profits, except for Japan, where Daiichi will retain exclusive rights.

As the price paid suggests, Enhertu is tipped to become a blockbuster, with peak sales predicted to exceed $4.5 billion annually. FDA approval is based on the results of the single-arm, pivotal phase 2 DESTINY-Breast01 trial of Enhertu (5.4 mg/kg) monotherapy in 184 female patients with HER2 positive metastatic breast cancer.
Trial results showed a confirmed objective response rate of 60.3%, including a 4.3% complete response rate and a 56.0% partial response rate.
A median duration of response of 14.8 months was demonstrated as of August 1, 2019. In addition, a median progression free survival of 16.4 months based on a median follow-up of 11.1 months, was recently reported at the San Antonio Breast Cancer Symposium and published online in The New England Journal of Medicine. Enhertu does have a boxed warning for interstitial lung disease pneumonitis (ILD) and embryo-foetal toxicity.

Safety of Enhertu has been tested in a pooled analysis of 234 patients with unresectable or metastatic HER2 positive breast cancer, based on data from DESTINY-Breast01 and a phase 1 trial. ILD occurred in 9% of patients, and deaths due to ILD and/or pneumonitis occurred in six patients – two deaths have already been reported from the phase 1 trial and four deaths in the phase 2 DESTINY-Breast01 trial.