Novartis announces positive phase III results showing effectiveness of BAF312 in patients with secondary progressive multiple sclerosis

Novartis Igea Luca Dezzani

August 25, 2016

Novartis has announced the results of the Phase III EXPAND study that evaluates the safety and efficacy of oral, once-daily, BAF312 (siponimod) in secondary progressive multiple sclerosis (SPMS). The study confirmed that BAF312 met its primary endpoint of a reduction in the risk of disability progression, compared with placebo. This EXPAND study represents the largest randomized, controlled study in SPMS to date.

Vasant Narasimhan, Global Head of Drug Development and Chief Medical Officer for Novartis, said: “SPMS is a particularly disabling form of MS, and there is a need for effective treatment options to help delay disability progression in those living with the condition. The positive EXPAND data are encouraging for a disease with such a high unmet need. We look forward to sharing the results at the upcoming ECTRIMS congress, and thank all of the study participants and investigators.”

Topline results of the EXPAND study, including primary and key secondary endpoints, will be presented at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), September 17th, in London, UK. In consultation with health authorities, Novartis will complete full analyses of the data and evaluate next steps.



FDA Approves TROXYCA® ER Extended-Release Capsules CII for Pain Management

Pfizer Igea Luca Dezzani

Pfizer Igea Luca Dezzani

August 19, 2016

Pfizer Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved TROXYCA® ER (oxycodone hydrochloride and naltrexone hydrochloride) extended-release capsules. These are intended for oral use, CII for the management of pain severe enough to require daily, around-the-clock, and for which alternative treatment options are insufficient. TROXYCA ER has properties that are expected to reduce abuse when crushed and administered by the oral and intranasal routes. Yet, abuse of TROXYCA ER by these routes is still possible.

Rory O’Connor, MD, Chief Medical Officer, Internal Medicine, Pfizer Inc., said: “Public health authorities and regulators have encouraged the development of treatments that are more difficult to abuse, yet offer pain relief to appropriate patients when used as indicated. The development of this medication with abuse-deterrent properties is another example of our ongoing commitment to advancing science and the treatment of patients with pain conditions.”

These TROXYCA ER extended-release capsules comprise pellets that consist of oxycodone hydrochloride. It is an opioid agonist which surround sequestered naltrexone hydrochloride, an opioid antagonist. The naltrexone is intended to remain sequestered when taken as directed, and patients receive oxycodone in an extended-release manner. Studies confirmed that when the pellets are crushed, the sequestered naltrexone is released and is available to counteract the effects of oxycodone.


Lilly And AstraZeneca Receive FDA Fast Track Designation For AZD3293 for Treating Early Alzheimer’s Disease


Aug 22, 2016

Eli Lilly and Company and AstraZeneca have announced that they have received Fast Track designation from U.S. Food and Drug Administration (FDA) for the development program in Alzheimer’s disease for AZD3293. It is an oral beta secretase cleaving enzyme (BACE) inhibitor currently in phase 3 clinical trials. The Fast Track program of FDA is designed to expedite the development and review of new therapies for treating serious conditions and tackle key unmet medical needs.

Phyllis Ferrell, vice president and global development leader for Alzheimer’s disease at Lilly, said: “We are pleased the FDA places a high priority on the development of drugs that target Alzheimer’s disease, one of the most critical health issues of our time. Most importantly, this is a positive step forward for the millions of patients, families, caregivers, advocates and healthcare providers who fight every day for progress.”

“The Fast Track designation in the U.S. for this promising potential therapy reinforces the ambition of the AstraZeneca-Lilly BACE alliance to help advance science for patients and their families managing this devastating illness. BACE inhibitors have the potential to transform the treatment of Alzheimer’s disease, one of the biggest challenges facing medical science today,” said Craig Shering, AZD3293 Project Lead in Global Medicines Development at AstraZeneca.

Top 10 Orphan Drugs and Their Economic Power

Orphan Drugs - Igea - Luca Dezzani

Rare diseases are known to bring significant impact on the quality of life of thousands to millions of people across the globe. Rare diseases are also referred to as orphan diseases because pharmaceutical companies refuse to adopt them to develop treatments. In fact, according to the National Center for Advancing Translational Sciences, there are approximately 7,000 rare diseases affecting around 25 to 30 million Americans. Unfortunately, there is still a long way to go to find solutions to end the suffering because treatment exists for only 200 to 300 orphan diseases and little is known about the cause of most rare diseases.

Yet, pharmaceutical companies oftentimes look at large disease populations as the main focus of their investment and show little interest in developing orphan drugs due to a number of reasons. One of these is that rare diseases only affect a small population, which equates to very low profitability. Furthermore, recruitment of patients for research and development (R&D) is also another challenge because it is difficult to locate patients and may eventually incur additional R&D costs. Fortunately, the industry has shifted its perception. In fact, some pharmaceutical companies are willing to take the risk of developing orphan drugs, which they now consider as potential money makers that can bring big revenue and a promising return on investment.

 Aside from this, to address the unmet treatment needs of various rare diseases, laws such as the Orphan Drugs Act of 1983 in the United States that gives financial incentives, were promulgated in order to encourage companies to venture on developing orphan drugs. Moreover, according to Thomson Reuters, other benefits given to drug companies for the R&D of orphan drugs are tax credits, favorable reimbursement, R&D grants, fewer hurdles to approval, waived FDA fees, longer exclusivity, shorter development timelines, lower marketing costs, greater regulatory success, faster uptake and premium pricing.

Though orphan drugs cater to a small patient pool, the high price of the drugs offsets the aforementioned challenge. In fact, in the year 2010, Soliris, which is known to treat paroxysmal nocturnal hemoglobinuria (PNH) that affects 1 out of 500,000, was actually considered as the industry’s most expensive drug amounting to $409,000 per year of treatment, which generated a total of $541 million revenue for Alexion Pharmaceuticals. In addition, in the year 2014, the top selling orphan drug in the USA in the year 2014 was Rituxan by Roche, a chronic lymphocytic leukemia drug, which yielded to $ 3.646 billion in total sales.

Development of orphan drugs can be one of the biggest breakthroughs in the pharmaceutical industry. It is projected that by the year 2018, the market will reach $127 billion or approximately 16% of the total prescription sales. Aside from the profitability associated with the commercialization of orphan drugs, patients with rare diseases will be given greater chances of surviving and hope as they face the battle against their illness.  The figure below will give us an overview of the top 20 orphan drugs that will have the projected highest revenue in 2018.

Orphan Drugs Infographics



Pfizer Announces Publication of New Analysis Showing the Effectiveness of Long-Term Therapy with VYNDAQEL (tafamidis) for the Treatment of Rare Neurodegenerative Disease

Pfizer Igea Luca Dezzani

Pfizer Igea Luca Dezzani

August 8, 2016

Pfizer Inc. has announced the publication of a new post-hoc analysis of data from three studies of VYNDAQEL in patients with mild transthyretin familial amyloid polyneuropathy (TTR-FAP). The analysis, which included patients with the Val30Met mutation treated over varying periods of up to 5.5 years, presented that treatment with VYNDAQEL started during the early stage of the disease resulted in minimal neurological disease progression. It also resulted in preservation of body weight, which frequently declines as the disease progresses. VYNDAQEL was well tolerated with no new safety signals observed. The new findings were published online in ‘Amyloid: The Journal of Protein Folding Disorders’.

TTR-FAP is a rare, progressive, genetic, and irreversible neurodegenerative disease that impairs quality of life significantly and is estimated to affect approximately 10,000 people worldwide. On average, people with TTR-FAP die within 10 years of symptom onset, when left untreated.

“These findings underscore the long-term benefits of early intervention with VYNDAQEL for symptomatic patients with TTR-FAP. This analysis, which is based on the longest prospective evaluation to date of any medication being studied for TTR-FAP, provides health care professionals with important insights into the management of patients with this disease,” said Dr. Kevin W. Williams, Chief Medical Officer, Rare Disease, Pfizer Innovative Health.

FDA Approves KEYTRUDA® (pembrolizumab) of Merck for Patients with Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

Merck Igea Luca Dezzani

Merck Igea Luca Dezzani

August 8, 2016

Merck has announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA®(pembrolizumab). It is the company’s anti-PD-1 (programmed death receptor-1) therapy, at a fixed dose of 200 mg every three weeks, for treating patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy. This indication for KEYTRUDA is approved under the FDA’s accelerated approval regulations, based on tumor response rate and durability of response.

Dr. Roger M. Perlmutter, president, Merck Research Laboratories, said: “Today’s approval represents a meaningful advance for the oncology community, as well as for our head and neck cancer clinical program. Together with prior approvals in the treatment of other tumor types, today’s action by the FDA underscores our tireless commitment to addressing the unmet needs of patients suffering from a broad range of cancers.”

“Head and neck cancer is a complex disease that historically has been associated with high recurrence rates and poor long-term outcomes, highlighting the critical need for new treatment options. The approval of KEYTRUDA for previously treated patients with recurrent or metastatic head and neck squamous cell carcinoma is an important step forward in treating this disease,” said Dr. Tanguy Seiwert, associate director of the Head and Neck Cancer Program and assistant professor of medicine at The University of Chicago.

Merck’s Phase 3 Study Results Evaluating ZEPATIER™ (elbasvir and grazoprevir) in Patients with Chronic Hepatitis C Receiving Treatment for Opioid Dependence Published

Merck Igea Luca Dezzani

Merck Igea Luca Dezzani

August 8, 2016

Merck has announced the publication of results from C-EDGE CO-STAR, which is a Phase 3 trial evaluating the use of ZEPATIER™ (elbasvir and grazoprevir) 50mg/100mg tablets in patients with chronic hepatitis C (HCV) genotype (GT) 1, GT4 and GT6 infection who receive opioid agonist therapy (OAT) (methadone and buprenorphine) that is normally used for the treatment of opioid addiction. The results were recently published online in the Annals of Internal Medicine, which indicated that treatment with 12 weeks of ZEPATIER resulted in high rates of sustained virologic response 12 weeks after the completion of therapy (SVR12, considered virologic cure based on undetectable HCV RNA levels).

Dr. Alain Litwin, professor of medicine and psychiatry and behavioral sciences at Albert Einstein College of Medicine, New York, said: “C-EDGE CO-STAR is the first phase 3 clinical trial dedicated to evaluating direct-acting antiviral therapy for chronic hepatitis C infection in patients on opioid agonist therapy without excluding patients actively using drugs with high abuse potential. This study demonstrates that people who inject drugs can be effectively treated with direct-acting antiviral therapy.”

“Merck continues to take a leadership role in exploring the potential to treat chronic hepatitis C infection in underserved and undertreated patient populations, including those who continue to use illicit drugs. These findings contribute to the robust body of evidence supporting the efficacy and safety profile of ZEPATIER in a broad range of patients with chronic hepatitis C genotype 1 or genotype 4 infection,” said Dr. Eliav Barr, vice president, infectious diseases, Merck Research Laboratories.

Takeda and Seattle Genetics Announce Positive Data from Phase 3 ALCANZA Clinical Trial of ADCETRIS® (Brentuximab Vedotin) for CD30-Expressing Cutaneous T-Cell Lymphoma


August 1, 2016

Takeda Pharmaceutical Company Limited and Seattle Genetics, Inc. have announced that the Phase 3 ALCANZA clinical trial assessing ADCETRIS (brentuximab vedotin) in patients with cutaneous T-cell lymphoma (CTCL) met its primary endpoint. It showed a highly statistically significant improvement in the rate of objective response lasting at least four months (ORR4).

Dirk Huebner, M.D., Executive Medical Director, Oncology Therapeutic Area Unit, Takeda Pharmaceutical Company, said: “These remarkable, clinically meaningful results from the completed ALCANZA trial represent an important milestone for the ADCETRIS program. If this new indication is approved by regulatory authorities, ADCETRIS may offer a novel treatment option for CTCL patients. We are excited by the data, which showed a significant improvement in the primary endpoint of ORR4 and all key secondary endpoints, along with a manageable safety profile. This outcome further establishes our commitment to patients living with CD30-expressing disease, and we look forward to sharing these data with regulatory authorities globally.”

“Cutaneous T-cell lymphoma is a debilitating, disfiguring and painful disease, and there is a significant need for additional effective treatment options with meaningful durable responses. This is the first Phase 3 randomized trial in CTCL versus an active control to read out, and we are thrilled to have successfully demonstrated the positive impact of using ADCETRIS for patients enrolled in this study. We anticipate reporting more complete ALCANZA data at the ASH annual meeting in December and intend to submit a supplemental Biologics License Application to the FDA in the first half of 2017 for approval in this setting,” said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics.

Takeda Gets Marketing Authorization for NINLARO™ (ixazomib) in Relapsed/Refractory Multiple Myeloma in Canada


August 8, 2016

Takeda Pharmaceutical Company Limited has announced Takeda Canada has received approval from Health Canada for NINLARO™ (ixazomib) capsules in combination with lenalidomide and dexamethasone for treating adult patients with multiple myeloma who have received at least one prior therapy. It is estimated that approximately 7,500 people live with multiple myeloma in Canada. The approval was mainly based on the results of the final analysis of the pivotal Phase 3 trial, TOURMALINE-MM1. It demonstrated that NINLARO in combination with lenalidomide and dexamethasone extended progression-free survival significantly, with a manageable safety profile in patients with relapsed/refractory multiple myeloma.

Chatrick Paul, General Manager at Takeda Canada, said: “Health Canada’s approval of NINLARO represents an important step in Takeda’s unwavering commitment to combat cancer by delivering novel therapies to patients as quickly, effectively and safely as possible. We are one of the first countries in the world to gain marketing approval to deliver NINLARO as a critical treatment option for multiple myeloma patients. We are pleased that NINLARO – our first oncology prescription medicine in Canada – has a product label that is broad and robust, meaning Canadians living with relapsed/refractory multiple myeloma will now have a new effective treatment option available to them in the comfort of their home.”

“Multiple myeloma, a devastating diagnosis for patients and their families, is a complicated disease that requires effective treatment options. The approval of NINLARO offers a much-needed new option for Canadian patients with multiple myeloma who have received at least one prior therapy. Its oral delivery may help multiple myeloma patients overcome some of the logistical burdens they may face with current therapies, which are typically administered in-clinic or in-hospital requiring significant travel and time constraints,” said Dr. Donna Reece, Professor and Director of the Program for Multiple Myeloma and Related Diseases in the Department of Medical Oncology and Haematology at Princess Margaret Hospital/University of Toronto.

GSK and Verily Agree to establish Galvani Bioelectronics – a new company dedicated to the development of bioelectronic medicines


01 August 2016

GSK has announced an agreement with Verily Life Sciences LLC (formerly Google Life Sciences) to form Galvani Bioelectronics in order to enable the research, development and commercialisation of bioelectronic medicines. In the new jointly owned company, GSK will hold a 55% equity interest and Verily will hold 45%. Galvani Bioelectronics will be headquartered in the UK, and the parent companies will contribute existing intellectual property rights and an investment of up to £540 million over seven years, subject to fruitful completion of various development and discovery milestones.

The agreement for establishing Galvani Bioelectronics signifies a vital next step in GSK’s bioelectronics research. The new company will bring together GSK’s world class drug discovery and development expertise and deep understanding of disease biology with Verily’s world leading technical expertise in the miniaturisation of low power electronics, device development, data analytics and software development for clinical applications.

Moncef Slaoui, GSK’s Chairman of Global Vaccines, said: “This agreement with Verily to establish Galvani Bioelectronics signals a crucial step forward in GSK’s bioelectronics journey, bringing together health and tech to realise a shared vision of miniaturised, precision electrical therapies. Together, we can rapidly accelerate the pace of progress in this exciting field, to develop innovative medicines that truly speak the electrical language of the body.”

“This is an ambitious collaboration allowing GSK and Verily to combine forces and have a huge impact on an emerging field.  Bioelectronic medicine is a new area of therapeutic exploration, and we know that success will require the confluence of deep disease biology expertise and new highly miniaturised technologies,” said Brian Otis, Verily’s Chief Technology Officer.