Archive | April, 2014

FDA Approves LDK 378

30 Apr

Chip and I are thrilled to have learned the Novartis trial drug he was on from March 28, 2013, to January 26, 2014, at Fox Chase Cancer Center in Philadelphia received breakthrough approval from the FDA yesterday. To think Chip was one of only 163 patients enrolled in this clinical trial is pretty astonishing.

We are so grateful for our proximity to Philadelphia from Washington, D.C., and that we had access to this trial and the means to travel every couple of weeks to receive this drug that provided him and our family with nine months of quality-filled days. To think Chip played a role in helping others who share the ALK+ genetic mutation to now have access to this drug is incredibly rewarding. You can read the related press release below.

Chip and I are also looking forward to attending a briefing in the Senate next week as guests of the Senate Special Committee on Aging, where the FDA Director, Janet Woodcock, will speak specifically about this drug. Details on the briefing follow.

– Sheila

Novartis gains FDA approval for Zykadia(TM), first therapy for patients with ALK+ NSCLC previously treated with the ALK inhibitor crizotinib

  • Zykadia (ceritinib) demonstrated an overall response rate of 54.6% in patients with ALK+ metastatic NSCLC who have no other treatment option[1]
  • Median duration of response to Zykadia was 7.4 months; patients in study started treatment with metastases, including brain (60%), liver (42%) and bone (42%)[1]
  • ALK+ NSCLC is driven by a rearrangement of the ALK gene, which is responsible for cancer cell growth in 2-7% of patients with NSCLC[2]
  • Approval follows FDA Breakthrough Therapy designation; regulatory application submitted in the EU and filings underway with other health authorities worldwide

Basel, April 29, 2014 – Novartis announced today that the US Food and Drug Administration (FDA) has approved Zykadia(TM) (ceritinib, previously known as LDK378) for the treatment of patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib[1]. The approval of Zykadia addresses an unmet medical need for patients with this type of lung cancer who have progressed on prior therapy.

“Zykadia represents an important treatment option for ALK+ NSCLC patients who relapse after starting initial therapy with crizotinib,” said lead investigator Alice T. Shaw, MD, PhD, Massachusetts General Hospital Cancer Center, Boston. “This approval will affect the way we manage and monitor patients with this type of lung cancer, as we will now be able to offer them the opportunity for continued treatment response with a new ALK inhibitor.”

Lung cancer is the leading cause of cancer death worldwide. The most common type of lung cancer is NSCLC, accounting for 85-90% of all cases[3]. Of those, 2-7% are driven by a rearrangement of the ALK gene, which increases the growth of cancer cells and can be identified by a molecular test of the cancer tumor[2]. Despite significant treatment advances for patients with ALK+ NSCLC, disease progression is often inevitable and more options are needed.

The approval of Zykadia is based on a pivotal trial that included 163 patients with metastatic ALK+ NSCLC who progressed on or were intolerant to treatment with crizotinib. The most common sites of metastases in the patient population studied were brain (60%), liver (42%) and bone (42%)[1].

Among previously-treated patients, Zykadia achieved an overall response rate (ORR) of 54.6% [95% CI, 47-62%] and a median duration of response (DOR) of 7.4 months [95% CI, 5.4-10.1 months][1]. The most common adverse reactions (incidence of at least 25%) were diarrhea, nausea, elevated transaminases, vomiting, abdominal pain, fatigue, decreased appetite and constipation[1].

“The approval of Zykadia less than three and a half years after the first patient entered our clinical trial exemplifies what is possible with a highly focused approach to drug development and strong collaboration,” said Alessandro Riva, MD, President, Novartis Oncology ad interim and Global Head, Oncology Development and Medical Affairs. “The dedication of clinical investigators, patients, the FDA and others has enabled us to bring this medicine to patients in need as swiftly as possible.”

Zykadia is an oral, selective inhibitor of ALK, an important therapeutic target in lung cancer. ALK is a gene that can fuse with other genes to form an aberrant “fusion protein” that promotes the development and growth of cancer cells[4],[5]. Zykadia is one of the first medicines to be approved following FDA Breakthrough Therapy designation, which was received in March 2013 due to the significance of results observed in the pivotal trial and the serious and life-threatening nature of ALK+ NSCLC. Additional regulatory submissions for Zykadia are underway worldwide, with an application currently filed in the European Union.

[Full press release with references]

Briefing on Breakthrough Therapies: Science and Progress at the FDA

A Friends of Cancer Research Congressional Briefing on the Progress of the FDA’s Breakthrough Therapies Program

Sponsored by:

Senator Michael Bennet (D-CO), Senator Orrin Hatch (R-UT), Senator Richard Burr (R-NC)

May 6, 2014 1:30pm-2:30pm 902 Hart Senate Office Building,


· Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research, US Food and Drug Administration (FDA)

· Ellen Sigal, Ph.D., Chair & Founder, Friends of Cancer Research

· Sandra Horning, M.D., Chief Medical Officer and Head of Global Product Development, Roche

· Urte Gayko, Ph.D., Senior Vice President of Global Regulatory Affairs, Pharmacyclics

· Moderator: Kate Rawson, Senior Editor, The RPM Report

Event Summary:

The FDA began implementing the Breakthrough Therapies Program in the summer of 2012, following the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA). After a speedy rollout, many stakeholders were quick to draw attention to the benefits that a Breakthrough Designation offers, namely, increased communication between drug sponsors and high-ranking FDA officials over the course of development, and an “all hands on deck” approach to drug review. In fact, in just two years, 178 requests for Breakthrough Designation have been submitted, 41 designations have been granted, and 5 drugs have been approved from the program.

In order to allow for the continued success and growth of the FDA’s Breakthrough Therapies Program, this briefing will convene expert stakeholders to explore key lessons learned and strategies for future application of this critical initiative.

· An hour-long briefing on Capitol Hill to evaluate the progress of the FDA’s Breakthrough Therapies Program, identifying how partnerships between government and industry have facilitated the development and review of the most urgently needed treatments.

· This briefing will also allow stakeholders to discuss lessons learned through participation in the program, including how Breakthrough Therapy designation has influenced the development of specific products.

· The briefing will aim to address how the Breakthrough Therapies Program can grow and evolve to encourage even more expeditious development and review of treatments that demonstrate significant improvements over existing options.