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May-June issue of JPPT is online
Posted by: Matthew Helms on Jun 13, 2017

The May June Issue of the Journal of Pediatric Pharmacology and Therapeutics is now available online.

Check it out at: http://jppt.org/toc/jppt/22/3​

FDA Expands Kalydeco (Ivacaftor) Indication to Treat Additional Mutations in Cystic Fibrosis Patients Age 2 Years and Older Using an Alternative to Human Clinical Studies
Posted by: Matthew Helms on May 23, 2017

On May 17, 2017, the U.S. Food and Drug Administration (FDA) expanded the approved indication for Kalydeco (ivacaftor) for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data. Kalydeco was previously approved for use in patients with one of ten different types of mutations in the CFTR, which occur in about 4 percent of CF patients. This approval expands the number of approved mutations from 10 to 33 and also provides a pathway to add additional rare CF mutations based on laboratory data.

The CFTR gene was identified almost 30 years ago and knowledge on the resultant CFTR channel’s structure and function, the clinical aspects of the disease and what causes it, and data on thousands of CF patients and their mutations have been accumulated. In addition, there is clear understanding of Kalydeco’s mechanism of action and a sufficient understanding of the risk/benefit profile for Kalydeco has been established from years of patient exposures for the previously approved mutations.

The expanded indication for Kalydeco was based on an alternative to human clinical studies, which are not feasible since many rare CF mutations have such small patient populations. Therefore, supporting information for the expanded indication relied on data from an in vitro cell-based model system built on Kalydeco’s existing knowledge base. Kalydeco’s ability to improve the function of the defective protein has been shown in a reliable in vitro cell model and was determined to reasonably accurately predict the likely response of patients with mutations not included in the initial clinical trials. Data from this model was deemed sufficient to determine whether certain populations with CF would likely respond to Kalydeco.

The recommended dose of KALYDECO for both adults and pediatric patients ages 6 years and older is one 150 mg tablet taken orally every 12 hours with fat-containing food. The recommended dose of KALYDECO (oral granules) for patients ages 2 to less than 6 years is 100 mg/day if body weight is less than 14 kg and 150 mg/day if body weight is 14 kg or greater administered just before or just after fat-containing food.

Common side effects of KALYDECO include headache; upper respiratory tract infection (common cold) including sore throat, nasal or sinus congestion, or runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness. KALYDECO is associated with risks including elevated liver transaminases and pediatric cataracts. Co-administration with strong CYP3A inducers (e.g., rifampin) substantially decreases exposure of KALYDECO, which may diminish effectiveness, and is therefore not recommended.


Full prescribing information is available at https://go.usa.gov/xNkCJ.

The FDA News Release on the expanded approved use of Kalydeco is available at https://go.usa.gov/xNkgg.

Visit Drugs@FDA at http://go.usa.gov/cMsjT for prescribing and patient information, approval letters, reviews and other information for FDA-approved drug products, which are often available shortly following approval.

A non-comprehensive list of examples of clinical substrates, inhibitors and inducers for metabolic and transporter system related interactions may be found at https://go.usa.gov/xXY9C.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at http://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178), or by mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).

The Office of Clinical Pharmacology (OCP) is pleased to offer the e-mail subscription service Clinical Pharmacology Corner. This is a free service from the FDA to provide occasional updates from OCP regarding newly approved therapies, new regulatory and scholarly publications, upcoming events, and other items of interest. Subscribe today (https://public.govdelivery.com/accounts/USFDA/subscriber/new?topic_id=USFDA_407).

The FDA always welcome your thoughts regarding the format, content, and utility of the communication. Comments may be sent via email to ocp@fda.hhs.gov.

This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.

PPAG Journal Accepted into Scopus
Posted by: Matthew Helms on May 22, 2017

PPAG is pleased to announce that the Journal of Pediatric Pharmacology and Therapeutics (JPPT) has been accepted into Scopus (https://www.elsevier.com/solutions/scopus). This is the largest abstract and citation database of peer-reviewed literature including both scientific journals, books and conference proceedings. Currently counting 22,794 peer-reviewed journals that delivers a comprehensive overview of the world's research output in the fields of science, technology, medicine, social sciences, and arts and humanities.

To be considered for review, all journal titles should meet all of these minimum criteria: 1) Consist of peer-reviewed content and have a publicly available description of the description of the peer review process peer review process; 2) Be published on a regular basis and have an International Standard Serial Number (ISSN) as registered with the ISSN International Centre; 3) Should in general have a 2 year publication history; 4) Have content that is relevant for and readable by an international audience, meaning: have references in Roman script and have English language abstracts and titles; and have a publicly available publication ethics and publication malpractice statement.

 

From APhA CEO blog: CDC recognizes growing need for physician–pharmacist partnerships
Posted by: Matthew Helms on May 18, 2017

Check out Thomas E. Menighan​ blog post here:

http://www.pharmacist.com/CEOBlog/cdc-recognizes-growing-need-physician-pharmacist-partnerships

 

Dr. Sarah Friebert (Our Garrett A. Helms Lecturer) Talks to PPAG
Posted by: Matthew Helms on May 6, 2017

Dr. Sarah Friebert, MD, Director of Pediatric Palliative Care at Akron Children's Hospital and our Inaugural Garrett A. Helms Memorial Lecturer, answers questions from the audience.

 

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