Breaking News
November 20, 2018 - Scientists identify novel target for neuron regeneration, functional recovery in spinal cord injury
November 20, 2018 - Skeletal imitation reveals how bones grow atom-by-atom
November 20, 2018 - Autism behaviors show unique brain network fingerprints in infants
November 20, 2018 - Location matters for inflammation clearance
November 20, 2018 - Towards finding a druggable cancer target
November 20, 2018 - Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Mid-2019
November 20, 2018 - Cooling ‘brains on fire’ to treat Parkinson’s
November 20, 2018 - Less pollution could increase the average lifespan of Copenhageners by an entire year in 2040
November 20, 2018 - Abramson Cancer Center becomes the 28th member institution of National Comprehensive Cancer Network
November 20, 2018 - The plug and play time-resolved spectrometer from PicoQuant
November 20, 2018 - Breakthrough technology offers new hope to people with glaucoma, retinitis and macular degeneration
November 20, 2018 - New report highlights key focus areas to help cancer screening realize its full potential
November 20, 2018 - International experts to discuss strategies to maintain spatial orientation in old age
November 20, 2018 - Scientists discover new inhibitor that decreases lung inflammation
November 20, 2018 - Participation project calls for relaxing research ban on germline interventions
November 20, 2018 - Karyopharm’s Selinexor Receives Fast Track Designation from FDA for the Treatment of Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma
November 20, 2018 - Arthritis by the Numbers: Book of Trusted Facts & Figures
November 20, 2018 - Drug homing method helps rethink Parkinson’s
November 20, 2018 - AHF commends the passage of global AIDS funding in the House, calls for swift approval
November 20, 2018 - The search for new psychiatric disorder treatments
November 20, 2018 - New research offers hope for simpler way to diagnose and treat cancer
November 20, 2018 - Study sheds light on the infection mechanism of influenza virus
November 20, 2018 - Storage failures of eggs and embryos gain a new perspective
November 20, 2018 - Buyers of short-term health plans: Wise or shortsighted?
November 20, 2018 - Study indicates that frogs in virus-exposed groups breed at young age
November 20, 2018 - FDA Alerts Health Care Professionals and Patients Not To Use Sterile Drug Products from Pharm D Solutions
November 20, 2018 - Asthma may contribute to childhood obesity epidemic
November 20, 2018 - Live probiotics can change existing gut flora and alter immune response
November 20, 2018 - Researchers to explore the enigmatic role of unstructured protein in regulating circadian function
November 20, 2018 - Many patients with adenomas do not receive colonoscopy within recommended time frame
November 20, 2018 - Drug used to treat PTSD does not reduce suicidal thinking, may worsen nightmares and insomnia
November 20, 2018 - In-person social contact may offer protection against depression and PTSD symptoms
November 20, 2018 - Routine HCV testing in correctional facilities can best identify and treat disease, say researchers
November 20, 2018 - Molecular DNA analysis could facilitate more accurate prognosis, treatment of aggressive brain tumors
November 20, 2018 - Breast Cancer Recurrence Rate Not Up With Autologous Fat Transfer
November 20, 2018 - Beta 2 Microglobulin (B2M) Tumor Marker Test: MedlinePlus Lab Test Information
November 20, 2018 - Could bariatric surgery make men more virile?
November 20, 2018 - Urine test to check if patients take their medications will save the NHS money, say researchers
November 20, 2018 - Study reveals impact of residual inflammatory risk on clinical outcomes after PCI
November 20, 2018 - RNAi therapy shown to alleviate preeclampsia
November 20, 2018 - Replacement of dysfunctional microglia has therapeutic potential for neurodegenerative diseases
November 20, 2018 - Forming 3D Neuronal Models of the Brain
November 20, 2018 - Shoulder ultrasounds could be used to predict diabetes
November 20, 2018 - SGLT2 Inhibitors for Diabetes Linked to Increased Risk for Amputation
November 20, 2018 - Stem cell transplant cements Arizona men’s father-son bond
November 20, 2018 - Scientists try to develop portable systems that can quickly produce biologics on demand
November 20, 2018 - Automating Data Capture and Image Analysis in Continuous Experiments
November 20, 2018 - New drug shows promise for treating people with peanut allergy
November 20, 2018 - Researchers develop novel mouse model to study immunomodulatory therapies
November 20, 2018 - “Britain must not go backward on antibiotic controls to appease US trade deals” – Jim Moseley, CEO of Red Tractor
November 20, 2018 - Widespread errors in ‘proofreading’ cause inherited blindness
November 20, 2018 - Reaping the benefits of living longer
November 20, 2018 - New Program Hopes to Make Early Detection and Treatment of ALS a Reality
November 19, 2018 - Artificial bone-like substance mimics the way real bone grows at atomic level
November 19, 2018 - FDA Grants Orphan Drug Designation To RGX-181 Gene Therapy For The Treatment Of CLN2 Form Of Batten Disease
November 19, 2018 - Systemic mastocytosis – Genetics Home Reference
November 19, 2018 - Eye trauma secondary to falls in older adults increasing
November 19, 2018 - Empowering women in India to improve their health: A Q&A
November 19, 2018 - Researchers have trained a computer to analyze breast cancer images and classify tumors
November 19, 2018 - New glucose binding molecule could be key to better metabolic control for diabetics
November 19, 2018 - Biologists uncover novel genetic control of lipid maintenance and its potential connection to lifespan
November 19, 2018 - Warmer winters may set scene for higher rates of violent crimes
November 19, 2018 - Personalized program of physical exercise effective in reversing functional decline in the elderly
November 19, 2018 - Acacia Pharma Resubmits Barhemsys New Drug Application
November 19, 2018 - PDL1 (Immunotherapy) Tests: MedlinePlus Lab Test Information
November 19, 2018 - Transforming pregnancy research with a smartphone app
November 19, 2018 - Stanford Medicine magazine explores how digital technology is changing health care
November 19, 2018 - Vision impairments may increase risk of falls in older adults
November 19, 2018 - Concomitant use of sleeping pills and opioids found to prevalent among people with Alzheimer’s disease
November 19, 2018 - Marijuana prevention programs should focus on promoting mental wellbeing of youth
November 19, 2018 - New report calls for greater awareness and emphasis on scale and impact of atrial fibrillation
November 19, 2018 - In throes of turkey salmonella outbreak, don’t invite illness to your table
November 19, 2018 - UK health policies should be redesigned to become more accessible for men
November 19, 2018 - Short Interpregnancy Intervals Tied to Adverse Outcome Risk
November 19, 2018 - New mothers’ breastfeeding pain can affect infant health
November 19, 2018 - Stanford Medicine magazine reports on ways digital technology is transforming health care | News Center
November 19, 2018 - Human drugs alter cricket personality
November 19, 2018 - Insilico Medicine to introduce ‘Cure a disease in a year’ program at Biodata World Congress 2018
November 19, 2018 - Experts debate over whether gut or brain is more important in regulating appetite
November 19, 2018 - Playing on fear and fun, hospitals follow pharma in direct-to-consumer advertising
FDA Approves Expanded Indication for Orkambi (lumacaftor/ivacaftor) in Children Ages 2-5 Years

FDA Approves Expanded Indication for Orkambi (lumacaftor/ivacaftor) in Children Ages 2-5 Years

image_pdfDownload PDFimage_print

BOSTON–(BUSINESS WIRE)–Aug. 7, 2018– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved Orkambi (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in this population. Orkambi oral granules are available in two dosage strengths (lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg) for weight-based dosing. Orkambi oral granules should be available for fulfillment within 2 to 4 weeks.

“For the first time, children ages 2 through 5 who have the most common form of CF have a treatment for the underlying cause of their disease,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We believe it is important to treat the underlying cause of the disease as early as possible and this approval is another significant milestone in our journey to bring effective medicines to all people living with CF.”

This FDA approval is based on a Phase 3 open-label safety study in 60 patients that showed treatment with Orkambi was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6 years and older. Improvements in sweat chloride, a secondary endpoint, were observed at week 24 (mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7, -27.6, n=49). Researchers also saw changes in key growth parameters, which were also secondary endpoints in the study. The most common adverse event (≥30%) was cough (63%); most adverse events were mild or moderate in severity. Four patients experienced serious adverse events (2 pulmonary exacerbations, 1 gastroenteritis, 1 constipation) and three patients discontinued treatment due to treatment emergent adverse events or elevated liver function tests. These findings were presented at the 41stEuropean Cystic Fibrosis Society Conference in June 2018.

“Cystic fibrosis is a systemic, multi-organ, progressive disease that is present from birth,” said Dr. John McNamara, Medical Director of the cystic fibrosis program at Children’s Minnesota hospital and lead study researcher. “Research suggests Orkambi could impact CF outcomes in patients as young as two years old. This approval is a significant development that enables physicians to begin treating the underlying cause of the disease in this population earlier than ever before.”

Orkambi was already approved in the U.S. for the treatment of CF in patients ages 6 and older who have two copies of the F508del-CFTR mutation. A Marketing Authorization Application (MAA) line extension for Orkambi in children ages 2 through 5 years has been submitted to the European Medicines Agency (EMA) with a decision anticipated in the first half of 2019.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

About Orkambi (lumacaftor/ivacaftor)

Orkambi is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex’s headquarters is now located in Boston’sInnovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry’s top places to work, including being named to Science magazine’s Top Employers in the life sciences ranking for eight years in a row.

For additional information and the latest updates from the company, please visit www.vrtx.com.

Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor), ORKAMBI®(lumacaftor/ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), VX-659 and VX-445 were discovered by Vertex as part of this collaboration.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including the statements by Dr. Kewalramani in the second paragraph and Dr. McNamara in the fourth paragraph of this press release and statements regarding (i) the timing of shipping of the oral granules in the United States and (ii) the anticipated timing of an EMA decision on the MAA line extension. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities may not approve, or approve on a timely basis, the company’s drug candidates due to safety, efficacy or other reasons, and the other risks listed under Risk Factors in Vertex’s annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex’s website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Source: Vertex Pharmaceuticals Incorporated

Posted: August 2018

Related Articles:

Orkambi (ivacaftor and lumacaftor) FDA Approval History

About author

Related Articles