Breaking News
May 21, 2018 - FDA Approves Aimovig (erenumab-aooe) as a Preventive Treatment for Migraine
May 21, 2018 - Infant death study reveals dangerous sleep practices among babysitters, relatives, others
May 21, 2018 - Intermittent fasting may increase diabetes risk, shows study
May 21, 2018 - New study shows declines in prostate cancer screening, diagnoses and treatment
May 21, 2018 - Fasting diets could raise risk of diabetes say experts
May 21, 2018 - FDA Alert: 7K and Poseidon 4500 by Shoreside Enterprises: Voluntary Recall
May 21, 2018 - Cell phones at summer camp: Research explores the effects
May 21, 2018 - Birth rate decline driven by waiting longer to have children, cost of infertility treatment
May 21, 2018 - In-hospital opioid prescribing may increase post-discharge opioid use, shows study
May 21, 2018 - ABPI expert urges to find new ‘blockbuster treatments’ for brain tumors
May 21, 2018 - Disruption of Circadian Rhythm Negatively Impacts Mental Health
May 21, 2018 - Researchers reveal mechanisms of periodic paralysis in people with rare genetic disorder
May 21, 2018 - World first use of cognitive training reduces gait freezing in Parkinson’s patients
May 21, 2018 - NIH stops alcohol study that was looking at purported health benefits of drinking
May 21, 2018 - Higher belly fat levels linked to greater risk of vitamin D deficiency
May 21, 2018 - Scientists collate evidence for mismatch between past evolutionary adaptation and modern lives
May 21, 2018 - New case report reveals negative clinical impact of using biotin supplement
May 21, 2018 - Researchers discover new disease mechanism in chronic tobacco smokers
May 21, 2018 - Breast Cancer Patients May Shorten Herceptin Regimen: Study
May 21, 2018 - Bias keeps women with higher body weights away from the doctor: study
May 21, 2018 - Researchers identify protein essential for eye lens development and clear vision
May 21, 2018 - Frontal cortical lesions moderate response to prism adaptation treatment after stroke
May 21, 2018 - Ultrasound guidelines can reliably differentiate between pediatric thyroid nodules that require biopsy
May 21, 2018 - Weight loss is an important predictor of cancer
May 21, 2018 - Ozone exposure at birth linked to increased risk of developing asthma in childhood
May 21, 2018 - CT scan still effective to determine thrombectomy treatment in stroke, study shows
May 21, 2018 - Clot busting drug combo reduces risk of major strokes in high risk patients
May 21, 2018 - New airway transplantation technique shows promising results in lung cancer patients
May 21, 2018 - Biomarker blood test does not appear to curb antibiotic overuse, shows new study
May 21, 2018 - Lilly’s Galcanezumab Meets Primary Endpoint in Phase 3 Study Evaluating Galcanezumab for the Prevention of Episodic Cluster Headache
May 21, 2018 - Grief symptoms similar in donor vs non-donor decision families
May 21, 2018 - Congo to start vaccinating populations against Ebola today to combat outbreak
May 21, 2018 - Researchers use MR spectroscopy to investigate mechanisms behind targeted treatment for gliomas
May 21, 2018 - Study reveals why older workers have higher stress levels than younger colleagues
May 21, 2018 - Health Tip: Taming a Pollen Allergy
May 21, 2018 - Inducing labor at 39 weeks reduces risks of C-section and other complications
May 20, 2018 - Developmental psychotherapy aims at helping antisocial adolescents become responsible adults
May 20, 2018 - People with OCD process emotions differently than their unaffected siblings
May 20, 2018 - Interfering with enzyme’s movement may be new approach for developing of anti-cancer drugs
May 20, 2018 - Prestroke and poststroke oral anticoagulation therapy in AF patients
May 20, 2018 - Why drug users prefer heroin at home, but cocaine while out
May 20, 2018 - Gene therapy that reverses blindness in dogs could also help treat humans
May 20, 2018 - Opioid-Related Payments Linked to Increase in Opioid Rx
May 20, 2018 - Phone apps push people to take their pills
May 20, 2018 - Backbreaking Work May Shorten Men’s Lives
May 20, 2018 - Harsher drug laws won’t stop violence, argues former police chief
May 20, 2018 - Cognitive decline in dementia is not reduced by exercise
May 20, 2018 - Detecting breast cancer with non-invasive ‘disease screening pill’
May 20, 2018 - Simple treatment may minimize hearing loss triggered by loud noises
May 20, 2018 - Alignment of mother and offspring body clock could prevent diseases such as heart disease and obesity
May 20, 2018 - New commercial data warehouse for life sciences
May 20, 2018 - Practice Intervention Targeting IV Opioids May Cut Exposure
May 20, 2018 - New study provides insight into blood signatures of inflammation
May 20, 2018 - Scientists make breakthrough discovery about vitamin B12
May 20, 2018 - What Causes Cancer? Misconceptions Abound
May 20, 2018 - Proper burial of dead cells limits inflammation
May 20, 2018 - Study offers novel solution to suppress metastatic spread of deadly breast cancer
May 20, 2018 - Perspectives of patients and caregivers on care transitions
May 20, 2018 - Guidelines for weight gain in pregnancy should be changed for underweight and very obese women
May 20, 2018 - Researchers transplant retinal sheets derived from human embryonic stem cells in retinal degeneration mouse models
May 20, 2018 - U.S. military personnel at greater risk for skin cancer than general population
May 20, 2018 - Your immune system holds the line against repeat invaders, thanks to this molecule
May 20, 2018 - Between death and deportation
May 20, 2018 - Developing a High Throughput Mass Spectrometry Platform for Drug Discovery
May 19, 2018 - New project aims to increase awareness among hospital clinicians of non-beneficial treatment at end-of-life
May 19, 2018 - Automated bone scan index offers accurate, speedy prognostic information about prostate cancer
May 19, 2018 - Rutgers Cancer Institute nurses research various topics to enhance patient experience
May 19, 2018 - Computer models provide valuable insight to structure and function of Ebola, Zika viruses
May 19, 2018 - Study exposes key tactic used by deadly fungus
May 19, 2018 - Bacterial signals are crucial to development of pre-leukemic myeloproliferation, study shows
May 19, 2018 - Global experts identify key issues in supporting children with brain injuries transition back to school
May 19, 2018 - Social connections may protect black men who have sex with men from acquiring HIV
May 19, 2018 - Study IDs Factors Linked to Quality of Life With Dementia
May 19, 2018 - Potassium — Consumer
May 19, 2018 - HIV-1 viruses transmitted at birth are resistant to antibodies in mother’s blood
May 19, 2018 - Some water pitchers are much better at removing toxins, shows research
May 19, 2018 - Scientists discover how unusually long strands of RNA help colon cancer cells avoid death
May 19, 2018 - International study finds viable treatment option for people with mild asthma
May 19, 2018 - Mayo discovery could enable development of personalized ovarian, brain cancer treatments
May 19, 2018 - ‘Superbug’ Surfaces at Poultry Farm in China
Shire Receives FDA Breakthrough Therapy Designation for Maribavir, an Investigational Treatment for Cytomegalovirus (CMV) Infection in Transplant Patients

Shire Receives FDA Breakthrough Therapy Designation for Maribavir, an Investigational Treatment for Cytomegalovirus (CMV) Infection in Transplant Patients

image_pdfDownload PDFimage_print

Cambridge, Mass. – January 4, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for maribavir (SHP620), a Phase 3 investigational treatment for cytomegalovirus (CMV) infection and disease in transplant patients resistant or refractory to prior therapy. By targeting a key CMV enzyme, maribavir is thought to inhibit CMV DNA replication and encapsidation, and prevent the escape of viral capsids from the nucleids of infected cells.

CMV is a beta herpes virus that, in patients with compromised immunity including organ or stem cell transplant recipients, causes clinically challenging complications that can be fatal. Existing antiviral therapies can be used to treat CMV, but their use may be limited by side effects and/or drug resistance.

“Maribavir has the potential to address critical medical needs for transplant patients who are refractory or resistant to currently available antiviral therapies, and I’m proud of the innovation and hard work that made this Breakthrough Designation Therapy milestone a reality,” said Andreas Busch, Global Head of R&D, Shire. “We are delighted that the FDA has granted Breakthrough Therapy Designation to maribavir and we continue to work every day to deliver therapies to the patients who need them most. We are eager to work with the FDA to continue development of maribavir.”

According to the FDA, Breakthrough Therapy Designation is granted to a therapy that is intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available or existing therapy. Under the designation, the FDA provides intensive guidance on an efficient drug development program, organizational commitment involving senior managers, and eligibility, based on supporting clinical data, for rolling and priority review of the marketing application; this process helps ensure patients have access to therapies as soon as possible, pending approval. Breakthrough Therapy Designation does not guarantee that FDA will approve maribavir for the treatment of CMV infections in transplant patients, and the timing of any such approval is uncertain.

Breakthrough Therapy Designation status for maribavir was granted based on the data from two Phase 2 studies, including a Phase 2 trial in 120 patients ages 12 or older with CMV infection. In the study, 67% of patients treated with varying doses of maribavir (400 to 1200 mg twice daily) for up to 24 weeks had no detectable levels of the virus in their blood plasma within six weeks of starting treatment. Dysgeusia (taste disturbance) was the most commonly reported treatment-emergent adverse event (AE) considered to be related to maribavir. Other related treatment-emergent AEs occurring in greater than or equal to 5% of study participants for any dose included nausea, CMV infection, immunosuppressant drug level increased, diarrhea, rash, vomiting, anaemia, and pruritus (itch).

About Maribavir

Maribavir, an investigational agent that is in a class of drugs called benzimidazole ribosides, is an orally bioavailable antiviral therapy being evaluated in patients with CMV infection after undergoing hematopoietic stem cell transplant or solid organ transplant. By inhibiting the CMV UL97 protein kinase, maribavir potentially affects several critical processes in CMV replication including viral DNA synthesis, viral gene expression, encapsidation and egress of mature capsids from the nucleus. Further research to confirm these findings will be conducted in pending Phase 3 studies.

The U.S. Food and Drug Administration (FDA) and the European Commission have granted Orphan Drug Designation to maribavir for treatment of clinically significant CMV viremia and disease in at-risk patients, and treatment of CMV disease in patients with impaired cell mediated immunity, respectively.

About CMV

CMV is a common virus that infects people of all ages. In the U.S., by age 40, over half of adults have been infected with CMV, most showing no signs or symptoms. However, in people with compromised immunity, CMV infection is a serious clinical complication that can lead to tissue-invasive disease and ultimately be fatal.

About Shire

Shire is the leading global biotechnology company focused on serving people with rare diseases. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.

Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.

Forward-Looking Statements

Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

Shire’s products may not be a commercial success;
increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire’s future revenues, financial condition and results of operations;
Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire’s products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire’s products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
the manufacture of Shire’s products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
certain of Shire’s therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
the actions of certain customers could affect Shire’s ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire’s revenues, financial conditions or results of operations;
Shire’s products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
adverse outcomes in legal matters, tax audits and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the Company’s revenues, financial condition or results of operations;
inability to successfully compete for highly qualified personnel from other companies and organizations;
failure to achieve the strategic objectives, including expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all with respect to Shire’s acquisitions, including NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated may adversely affect Shire’s financial condition and results of operations;
Shire’s growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to Shire’s reputation, the withdrawal of the product and legal action against Shire;
investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire’s activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which may decrease its business flexibility and increase borrowing costs; and
a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM 1A: Risk Factors”, and in Shire’s subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

Source: Shire plc

Posted: January 2018

Tagged with:

About author

Related Articles