Breaking News
February 21, 2018 - Father’s stress affects the brain development of offspring, mice study shows
February 21, 2018 - ESRD Death Declines in Vasculitis Patients
February 21, 2018 - Taking ibuprofen for long periods found to alter human testicular physiology
February 21, 2018 - Google AI device could predict a person’s risk of a heart attack
February 20, 2018 - FDA Approves Domestic Source for Tc-99m Isotopes
February 20, 2018 - Sanofi rejects refund demand faces Philippine suit over dengue vaccine (Update)
February 20, 2018 - Researchers discover that activation of specific enzyme may help suppress tumor metastasis
February 20, 2018 - Blood or marrow transplantation survivors have higher risk of cognitive impairment
February 20, 2018 - Booze Beats Pot at Being Unhealthy: Oregon Poll
February 20, 2018 - Morning Break: ’20 Years Late’; Drugs in the Dirt; Catching Flu in the Dorm
February 20, 2018 - Another piece to the puzzle in naked mole rats’ long, cancer-free life
February 20, 2018 - Scientists identify four viruses that can produce insulin-like hormones
February 20, 2018 - New e-Health solution developed to prevent cardiovascular disease, dementia in senior citizens
February 20, 2018 - New genetic risk score could help guide screening decisions for prostate cancer
February 20, 2018 - Study finds higher risk of stroke among blacks with atrial fibrillation than whites
February 20, 2018 - Physical activity could be used as strategy for diabetes prevention
February 20, 2018 - Researchers develop sensing method for early detection of cancer and diabetes
February 20, 2018 - New wearable electronics could be game-changer for stroke rehabilitation
February 20, 2018 - Immune history influences person’s response to flu vaccine
February 20, 2018 - Serenity Now! Learn to Have Patience with Patients
February 20, 2018 - Computer simulation addresses the problem of blood clotting
February 20, 2018 - Women with type 1 diabetes not protected against coronary artery disease
February 20, 2018 - Persistent bloating can be a sign of ovarian cancer, warns charity
February 20, 2018 - Trump administration proposes rule to loosen curbs on short-term health plans
February 20, 2018 - Key protein involved in epigenetic regulation of gene expression guides skin cell renewal
February 20, 2018 - Heart attack symptoms often missed in women
February 20, 2018 - Diagnosis of celiac disease takes 3.5 years for patients who do not report GI symptoms
February 20, 2018 - Study reveals functional dynamics of ion channels
February 20, 2018 - Study explores link between mortality risk and combustible tobacco use
February 20, 2018 - ‘She Trusted Me, and I’d Turned Her Away’
February 20, 2018 - AbbVie and Voyager Therapeutics collaborate to develop new treatments for tauopathies
February 20, 2018 - Fast food makes the immune system more aggressive in the long term
February 20, 2018 - Therapeutic target for glaucoma could have treatment ramifications for Alzheimer’s and Parkinson’s
February 20, 2018 - Overcoming Negative Reviews | Medpage Today
February 20, 2018 - MyD88—villain of allergies and asthma
February 20, 2018 - Food scientists develop rapid screening technique to detect pesticide residue in vegetables
February 20, 2018 - Lab-grown cerebellar cells may help explain how ASD develops at molecular level
February 20, 2018 - Scientists explore connection between bad sleep habits and stiff blood vessels
February 20, 2018 - New Treatment Apalutamide (Erleada) Approved for Prostate Cancer That Resists Hormone Therapy
February 20, 2018 - Do You Really Need My Signature on That?
February 20, 2018 - HIV-1 genetic diversity is higher in vaginal tract than in blood during early infection
February 20, 2018 - Diabetes does not increase work-loss years due to early retirement
February 20, 2018 - Researchers aim to find out how PTSD affects decisions of police
February 20, 2018 - UH Cleveland Medical Center explores novel treatments for uterine fibroids
February 20, 2018 - Flu Vax Efficacy 25% Against Predominant H3N2 Strain So Far
February 20, 2018 - HIV screening most optimal at 25 years of age if no risk factors
February 20, 2018 - Loyola Medicine primary care physician offers advice to minimize risk of flu
February 20, 2018 - Safe sleep recommendations for parents that may help reduce child’s risk of SUID
February 20, 2018 - Why Do So Few Docs Have Buprenorphine Waivers?
February 20, 2018 - Low levels of alcohol good for the brain
February 20, 2018 - Experimental treatment improves invisible symptoms of a man with spinal cord injury
February 20, 2018 - Myriad’s EndoPredict offers better prediction of breast cancer recurrence, analysis shows
February 20, 2018 - Researchers identify fifteen genes that determine our facial features
February 20, 2018 - Morning Break: New Health IT Player; Luxturna No Bargain; Nuclear Freakout
February 20, 2018 - How does it compare? Hospice care at home, at assisted living facility, at nursing home
February 19, 2018 - Scientists develop water-soluble warped nanographene for bioimaging
February 19, 2018 - It’s Not Your Imagination: You’re Hungrier After Losing Weight
February 19, 2018 - Antihypertensive Use At Delivery Rising in Preeclampsia
February 19, 2018 - A centuries-old math equation used to solve a modern-day genetics challenge
February 19, 2018 - Liquid biopsies could be used as new predictive marker for metastatic TNBC
February 19, 2018 - Russian researchers develop new multi-layered biodegradable scaffolds
February 19, 2018 - Are ‘Vaccine Skeptics’ Responsible for Flu Deaths?
February 19, 2018 - Hidden genetic effects behind immune diseases may be missed, study suggests
February 19, 2018 - Emergency nurses experience regular verbal and physical abuse
February 19, 2018 - Study sheds light on biology that guides behavior across different stages of life
February 19, 2018 - Morning Break: Transgender Breast Feeding; Brazilian ‘Pro-Vaxxers’; Post-Stroke Exercise
February 19, 2018 - Meningitis vaccination strategy in Africa found to be effective, economical
February 19, 2018 - Researchers uncover how excess calcium may influence development of Parkinson’s disease
February 19, 2018 - Psoriasis drug also effective at reducing aortic inflammation
February 19, 2018 - Excess emissions can make serious contributions to air pollution, study shows
February 19, 2018 - Researchers reveal potential biological roots behind individuality
February 19, 2018 - Diabetes Drugs Differ on HF; School-Based Obesity Program Flop; Plaque Type in ACS
February 19, 2018 - Surgical infections linked to drug-resistant bugs, study suggests
February 19, 2018 - Poor awareness may hinder a child’s early dental care
February 19, 2018 - Research finds rising trend in incidence of merkel cell carcinoma
February 19, 2018 - Researchers uncover Ras protein’s role in uncontrolled cancer growth
February 19, 2018 - FDA Approves Apalutamide (Erleada) to Help Curb a Tough-to-Treat Prostate Cancer
February 19, 2018 - Educational Tool Boosts Cervical Length Screening
February 19, 2018 - Spider’s web inspires removable implant that may control type 1 diabetes
February 19, 2018 - Scientists develop fluorescent probe to identify cancer stem cells
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