Breaking News
January 18, 2019 - Let’s map our DNA and save billions each year in health costs
January 18, 2019 - AI demonstrates potential to identify irregular heart rhythms as well as humans
January 17, 2019 - Study shows link between air pollution and increased risk of sleep apnea
January 17, 2019 - Neck-strengthening exercises can protect athletes from concussions
January 17, 2019 - Computer model shows how to better control MRSA outbreaks
January 17, 2019 - Pain is unpleasant, and now scientists have identified the set of responsible neurons
January 17, 2019 - CUIMC Celebrates 2018-2019
January 17, 2019 - Study reveals potential pathway for endothelial cells to avoid apoptosis
January 17, 2019 - Hamilton Storage launches LabElite DeCapper SL to expand LabElite product family
January 17, 2019 - Location of epigenetic changes co-locate with genetic signal causing psychartric disorder
January 17, 2019 - Researchers awarded 6.1 million euros to address female fertility problems
January 17, 2019 - Counseling appointments fail to reduce weight gain during pregnancy, shows study
January 17, 2019 - Contraceptive patch that could provide 6 months of contraception within seconds
January 17, 2019 - Yeast model may pave way for development of novel therapies for metabolic disorders
January 17, 2019 - Study determines impact of antibiotic perturbation of the gut microbiome on skeletal health
January 17, 2019 - Cardiometabolic Risk Up With Tourette, Chronic Tic Disorder
January 17, 2019 - Hong Kong scientists claim ‘broad-spectrum’ antiviral breakthrough
January 17, 2019 - Researchers discover the brain cells that make pain unpleasant | News Center
January 17, 2019 - Hepatitis Is Common in New Cancer Patients
January 17, 2019 - Podcast: KHN’s ‘What The Health?’ Drug Prices Are Rising Again. Is Someone Going To Do Something About It?
January 17, 2019 - Smoking significantly increases your biological age, study shows
January 17, 2019 - B-group vitamins may be beneficial for people with first episode psychosis
January 17, 2019 - Researchers demonstrate how manganese produces parkinsonian syndrome
January 17, 2019 - Researchers suggest link between personality type and attitude towards others’ bodies
January 17, 2019 - Mutant mice administered with cocaine failed to exhibit hyperactivity, shows study
January 17, 2019 - Health Tip: Understanding a Heart Murmur
January 17, 2019 - Gut protein mutations shield against spikes in glucose
January 17, 2019 - Engineered immune cells target broad range of pediatric solid tumors in mice | News Center
January 17, 2019 - Study finds link between high pesticide exposure and poor sense of smell among farmers
January 17, 2019 - Many cancer patients have undiagnosed hepatitis
January 17, 2019 - New study finds only 13% of outpatient antibiotic prescriptions to be appropriate
January 17, 2019 - Stem cell-based approach to diabetes offers hope for treatment
January 17, 2019 - New project receives €8.65 million from EU and Canada to ease genomic, health data sharing
January 17, 2019 - Improvements in pharmacological study to fight cognitive impairment in schizophrenia
January 17, 2019 - Study looks at trends over time in oral antibiotic prescribing by dermatologists
January 17, 2019 - Most substance use disorder treatment facilities do not offer medication treatment
January 17, 2019 - Multiple sclerosis could benefit from stem cell therapy
January 17, 2019 - Researchers manipulate T cells to improve transplant success
January 17, 2019 - Put away your rulers and reach for your phone
January 17, 2019 - Mindfulness linked with fewer menopausal symptoms
January 17, 2019 - Integrated care to women with PMADs offered at several levels
January 17, 2019 - Researchers identify MANF as a rejuvenating factor in parabiosis
January 17, 2019 - Truncal mutations study suggests new direction in origins of cancer
January 17, 2019 - Beckman Coulter launches new ClearLLab 10C System for clinical flow cytometry lab
January 17, 2019 - Effects of linoleic acid on the body are largely dependent on genes, shows study
January 17, 2019 - Pre-injury exercise reduces damage to both muscles and nerves, study finds
January 17, 2019 - Minimizing Antibody Size to Maximize Research Potential
January 17, 2019 - Research finds large genome in tiny forest defoliator
January 17, 2019 - Technology helps reduce the yearning for unhealthy food
January 17, 2019 - Imec develops prototype cardiovascular device
January 17, 2019 - New Drug Application for Insomnia Disorder Treatment Lemborexant Submitted in the United States
January 17, 2019 - What you should know about teeth whitening
January 17, 2019 - Why Older Adults Should Eat More Protein (And Not Overdo Protein Shakes)
January 17, 2019 - Colorectal cancer mortality rates predicted to increase globally
January 17, 2019 - Scientists discover mutational signatures of tumor hypoxia
January 17, 2019 - New evidence shows how fever alters immune cells
January 17, 2019 - Researchers find new class of blood pressure-regulating peptides in vampire bat venom
January 17, 2019 - Promega to exhibit new Maxwell RSC48 platform at 2019 Festival of Genomics
January 17, 2019 - Study pinpoints immune cells that could be key to tackling hypertension
January 17, 2019 - Couples Intervention May Aid Partners of Diabetes Patients
January 17, 2019 - Your weight history may predict your heart failure risk
January 17, 2019 - Explore a cornucopia of accomplishments in prematurity research
January 17, 2019 - New study identifies four characteristics that predict severity of postpartum depression
January 17, 2019 - AHF urges United Nations to follow own mandate for protecting Ebola response efforts
January 17, 2019 - New, scalpel-free treatment for reducing Parkinson’s tremor gets FDA approval
January 17, 2019 - Neurobiologists uncover key component of how the human brain marks time
January 17, 2019 - LifeTime receives fund to develop a plan to embed its vision for healthier future
January 17, 2019 - WTC first responders at higher risk for head and neck cancers, study finds
January 17, 2019 - New NSF funded study may help physicians decrease brain injury deaths
January 17, 2019 - Ham bones contain peptides that could have cardioprotective effects
January 17, 2019 - Research finds how Candida albicans adapt to low oxygen levels to cause infection
January 17, 2019 - Cobra Biologics announces appointment of Dr Darrell Sleep as Director of Innovation
January 17, 2019 - Cellular protein that interacts with viruses appears to enable infection process of Zika virus
January 17, 2019 - Opioids Now More Deadly for Americans Than Traffic Accidents
January 17, 2019 - Women who start periods early are at greater risk of cardiovascular problems
January 17, 2019 - The brain-circuitry clash that keeps you from diving into that plate of ribs when you’re dining with royalty
January 17, 2019 - Poo transplant can successfully treat patients with ulcerative colitis
January 17, 2019 - Study suggests key role for glial cells in Parkinson’s disease
January 17, 2019 - Educational videos in clinical settings increase HPV vaccination rates among adolescents
January 17, 2019 - Better understanding of aggressive brain tumour
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