Top Pharma News – September and October, 2016

 

Bristol-Myers Squibb’s ORENCIA® (abatacept) is Approved by European Commission for the Treatment of Adult Patients with Rheumatoid Arthritis

Bristol-Myers

September 6, 2016

Bristol-Myers Squibb Company has announced that the European Commission has approved ORENCIA ® (abatacept) intravenous (IV) infusion and subcutaneous (SC) injection, combined with methotrexate (MTX), for treating highly active and progressive disease in adult patients with rheumatoid arthritis (RA) not previously treated with MTX. Through this approval, ORENCIA has turned out as the first biologic therapy with an indication in the European Union (EU) specifically applicable to the treatment of MTX-naive RA patients with highly active and progressive disease. This approval permits the expanded marketing of ORENCIA in all 28 Member States of the EU.

FDA Accepts Supplemental Biologics License Application for Merck’s KEYTRUDA® (pembrolizumab) for First-Line Treatment of Patients with Advanced Non-Small Cell Lung Cancer

Merck Igea Luca Dezzani

September 7, 2016

Merck has announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for KEYTRUDA® (pembrolizumab) for Priority Review.  KEYTRUDA® is Merck’s anti-PD-1 therapy that is used as the first-line treatment of patients with advanced non-small cell lung cancer (NSCLC). In addition, the FDA granted Breakthrough Therapy Designation for this indication. Merck has also submitted a Marketing Authorization Application to the European Medicines Agency for KEYTRUDA®.

Janssen Submits Marketing Authorisation Application to European Medicines Agency for Darunavir-Based Single Tablet Regimen for the Treatment of HIV-1

janssen-logo

September 12, 2016

Janssen-Cilag International NV (Janssen) has announced that it has submitted a Marketing Authorisation Application to the European Medicines Agency (EMA), which seeks approval for a new once-daily darunavir-based single tablet regimen (STR). This tablet would be the first protease inhibitor (PI)-based STR option (D/C/F/TAF FDC) if approved, indicated as a complete regimen for the treatment of human immunodeficiency virus type 1 (HIV‑1) infection in adults and adolescents (aged 12 years and older with body weight of at least 40 kg).

Janssen submits application to European Union seeking approval of sirukumab for rheumatoid arthritis

janssen-logo

September 12, 2016

Janssen-Cilag International NV (Janssen) has announced the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of sirukumab for treating adult patients with moderately to severely active rheumatoid arthritis (RA). Approximately 6.2 million Europeans are affected by RA, which is a chronic, systemic inflammatory condition. Sirukumab is a human monoclonal IgG1 kappa antibody targeting the cytokine IL-6, a naturally occurring protein that is believed to play a key role in autoimmune conditions like RA.

Merck and Pfizer Announce Investigational Ertugliflozin Met Primary Endpoint of A1C Reduction When Added to Metformin and Sitagliptin for the Treatment of Type 2 Diabetes

merck-pfizer

September 15, 2016

Merck, in partnership with Pfizer Inc., has announced that a Phase 3 study (VERTIS SITA2) of ertugliflozin, an investigational oral SGLT2 inhibitor for the treatment of patients with type 2 diabetes, met its primary endpoint. Both 15 mg and 5 mg daily doses of ertugliflozin revealed meaningfully greater reductions in A1C of 0.69 percent and 0.76 percent, respectively, in comparison with placebo (p<0.001, for both comparisons), when added to patients on a background of stable metformin (≥1500 mg/day) and sitagliptin (100 mg/day). For the first time, these study results were presented during an oral session at the 52nd Annual Meeting of the European Association for the Study of Diabetes (EASD) in Munich, Germany.

Phase III combination trial of Bydureon and Forxiga shows significant benefits in patients with type-2 diabetes

astra

September 16, 2016

Positive results from the Phase III DURATION-8 trial confirmed that Bydureon (exenatide extended-release formulation) 2mg once weekly, combining with Forxiga (dapagliflozin) 10mg once daily significantly reduced blood sugar as measured by HbA1c, versus the individual medicines alone in patients with type-2 diabetes ineffectively controlled on metformin. The results were presented today at the 52nd Annual Meeting of the European Association for the Study of Diabetes (EASD) in Munich, Germany, and simultaneously published in the journal ‘The Lancet Diabetes & Endocrinology’.

Pfizer Gets Positive CHMP Opinion for IBRANCE® (palbociclib) in Combination with Endocrine Therapy for The Treatment of HR+/HER2- Metastatic Breast Cancer in Europe

Pfizer Igea Luca Dezzani

September 16, 2016

Pfizer Inc. has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending that IBRANCE® (palbociclib) be granted marketing authorization in the European Union (EU) for treating women with hormone receptor-positive, human epidermal growth factor receptor 2-negative (HR+/HER2-) locally advanced or metastatic breast cancer. This positive opinion from CHMP is for IBRANCE to be used in combination with fulvestrant in women who have received prior endocrine therapy, as well as in combination with an aromatase inhibitor. European Commission (EC) will now review The CHMP’s opinion.

U.S. FDA Accepts Biologics License Application for Romosozumab, Amgen and UCB Announce

amgen-ucb

September 26, 2016

Amgen and UCB have announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for romosozumab. It is an investigational monoclonal antibody for treating osteoporosis in postmenopausal women at increased risk of fracture. Romosozumab works by binding and hindering the activity of the protein sclerostin, naturally occurring in the bone, thereby reducing bone resorption and increasing bone formation.

Amgen Announces Top-Line Results from Phase 3 KYPROLIS® (Carfilzomib) CLARION Study in Patients with Newly Diagnosed Multiple Myeloma

Amgen Igea Luca Dezzani

September 27, 2016

Amgen has announced top-line results of the Phase 3 CLARION trial evaluating an investigational regimen of KYPROLIS® (carfilzomib), melphalan and prednisone (KMP) versus Velcade® (bortezomib), melphalan and prednisone (VMP) for 54 weeks in patients with newly diagnosed multiple myeloma who were not eligible for hematopoietic stem-cell transplant. The trial did not meet the primary endpoint of superiority in progression-free survival (PFS). The observed hazard ratio (KMP versus VMP) was 1.21 (95 percent CI, 0.90 – 1.64) while the data for overall survival, a secondary endpoint, are not yet mature. Neither result was statistically significant. These data will be submitted to a future medical conference and for publication.

Two Cardiovascular Collaborations Announced by Amgen and Arrowhead Pharmaceuticals

amgen-arrowhead

September 29, 2016

Amgen and Arrowhead Pharmaceuticals Inc. have announced two license and collaboration agreements to develop and commercialize RNA interference (RNAi) therapies for cardiovascular disease. Under the first agreement, Amgen receives a worldwide, exclusive license to Arrowhead’s novel, RNAi ARC-LPA program. The RNAi molecules are designed to decrease elevated lipoprotein, which is a genetically validated, independent risk factor for atherosclerotic cardiovascular disease. As per the second agreement, Amgen receives an option to a worldwide, exclusive license for a RNAi therapy for an undisclosed genetically validated cardiovascular target. Amgen will be wholly responsible for clinical development and commercialization in both agreements.

Treatment outcome data from The Prostate Cancer Registry presented for the first time at the 2016 ESMO Congress

janssen-logo

October 10, 2016

Janssen-Cilag International NV has presented the first reported primary treatment outcome data from The Prostate Cancer Registry at the 2016 European Society for Medical Oncology (ESMO) Congress in Copenhagen, Denmark. The Prostate Cancer Registry is the first and largest prospective study of men with metastatic castration-resistant prostate cancer (mCRPC) in Europe. The preliminary data suggest that chemotherapy-naïve patients benefit more from treatment than post-chemotherapy patients. Furthermore, patients have a higher prostate-specific antigen (PSA) response when treated with androgen receptor-targeted agents than with taxanes, after first line docetaxel treatment.

Pfizer Presented New Data on XELJANZ® for Ulcerative Colitis at UEG Week 2016

Pfizer Igea Luca Dezzani

October 15, 2016

Pfizer Inc. announced that three abstracts for XELJANZ® (tofacitinib citrate), being investigated in moderate to severe ulcerative colitis (UC), were presented at the United European Gastroenterology Week (UEG Week 2016), October 15-19 in Vienna, Austria. The tofacitinib presentations highlighted new research results from the Phase 3 Oral Clinical Trials for tofAcitinib in ulceratiVE colitis (OCTAVE) Induction trials, including one oral presentation looking at the impact of prior treatment with tumor necrosis factor inhibitors (TNFi) on efficacy endpoints. Furthermore, two abstracts were accepted as poster presentations, highlighting results by endoscopic response, and onset of action, respectively.

 

Amgen Announces Positive Top-Line Results From XGEVA® (Denosumab) Phase 3 Trial for Patients with Multiple Myeloma

Amgen Igea Luca Dezzani

October 20, 2016

Amgen has announced that a Phase 3 study evaluating XGEVA® (denosumab) versus zoledronic acid met the primary endpoint of non-inferiority (hazard ratio = 0.98, 95 percent CI, 0.85 – 1.14) in delaying the time to first on-study skeletal-related event (SRE) in patients with multiple myeloma. The secondary endpoints of superiority in delaying time to first SRE and delaying time to first-and-subsequent SRE were not met. XGEVA’s hazard ratio versus zoledronic acid for overall survival was 0.90 (95 percent CI, 0.70 – 1.16).

NICE Recommends Oral OTEZLA® (Apremilast) for Adults with Chronic Plaque Psoriasis

celgene

October 20, 2016

Celgene has announced that adult patients in England and Wales with chronic plaque psoriasis will now have access to oral OTEZLA ® (apremilast) after a positive final appraisal determination from the National Institute for Health and Care Excellence (NICE). The decision is the conclusion of a NICE Rapid Review. It ensures patients in England and Wales will join those in Scotland, who have been benefitting from access to OTEZLA since Scottish Medicines Consortium (SMC) recommended it in June 2015. It has been estimated that Psoriasis has affected around 960,000 adults in the UK, seriously impacting their daily lives.

NICE Recommends Oral OTEZLA® (Apremilast) for Adults with Chronic Plaque Psoriasis

celgene

October 20, 2016

Celgene has announced that adult patients in England and Wales with chronic plaque psoriasis will now have access to oral OTEZLA ® (apremilast) after a positive final appraisal determination from the National Institute for Health and Care Excellence (NICE). The decision is the conclusion of a NICE Rapid Review. It ensures patients in England and Wales will join those in Scotland, who have been benefiting from access to OTEZLA since Scottish Medicines Consortium (SMC) recommended it in June 2015. It has been estimated that Psoriasis has affected around 960,000 adults in the UK, seriously impacting their daily lives.

“NICE’s decision to recommend apremilast for the treatment of psoriasis is an important step forward in the management of a disease which for many patients can have a significant detrimental effect on their lives. Apremilast offers patients a much needed new oral treatment option that does not require routine laboratory monitoring. Clinical trials of apremilast demonstrated a reduction in severity of psoriasis and associated itching as well as improvement in hard to treat areas, such as the nails and scalp. The drug has the potential to fill an important gap in the psoriasis treatment pathway and its introduction is welcomed by patients and healthcare practitioners,” commented Professor Chris Griffiths, Professor of Dermatology, University of Manchester.

 

U.S. FDA Approves Vermox™ Chewable (Mebendazole) for Treating both Children and Adults with Whipworm and Roundworm Infections

janssen-logo

Oct 19, 2016

Janssen Pharmaceuticals, Inc. has announced that the Food and Drug Administration (FDA) of U.S. has approved VERMOXTM  CHEWABLE (mebendazole chewable 500mg tablets) for treating patients, both adults and children, with gastrointestinal infections caused by Trichuris trichiura (whipworm) and Ascaris lumbricoides (roundworm). Around the world, this new tablet formulation can provide alternative treatment for millions of children infected by intestinal worms.

“Roundworm and whipworm are among the most prevalent forms of intestinal parasitic infections afflicting children in the world today, particularly in vulnerable and neglected communities. The FDA approval of VERMOXTM CHEWABLE represents an important milestone in our efforts to develop a new therapy that safely cures patients with intestinal infections by roundworm and whipworm. We will work with the World Health Organization (WHO), other health authorities and partners to ensure VERMOXTM CHEWABLE is available as part of our longstanding global product donation program,” said Paul Stoffels, M.D., Chief Scientific Officer, Johnson & Johnson.

Dr. David Addiss, Director of Children Without Worms (CWW), a partnership between Johnson & Johnson, GSK and The Task Force for Global Health, said: “Roundworm and whipworm infect millions of children, most without reliable access to safe drinking water and good sanitation. A chewable form of mebendazole fills a major gap in global treatment efforts against intestinal worms and helps us treat children when they are most vulnerable to these debilitating infections.”

Amgen Announces Positive Top-Line Results From XGEVA® (Denosumab) Phase 3 Trial for Patients with Multiple Myeloma

Amgen Igea Luca Dezzani

Amgen Igea Luca Dezzani

Oct. 20, 2016

Amgen has announced that a Phase 3 study evaluating XGEVA® (denosumab) versus zoledronic acid met the primary endpoint of non-inferiority (hazard ratio = 0.98, 95 percent CI, 0.85 – 1.14) in delaying the time to first on-study skeletal-related event (SRE) in patients with multiple myeloma. The secondary endpoints of superiority in delaying time to first SRE and delaying time to first-and-subsequent SRE were not met. XGEVA’s hazard ratio versus zoledronic acid for overall survival was 0.90 (95 percent CI, 0.70 – 1.16).

Adverse events observed in patients treated with XGEVA were generally consistent with its known safety profile. Nausea and diarrhea were the most common adverse events (greater than 25 percent) in the XGEVA arm of the study.

Sean E. Harper, M.D., executive vice president of Research and Development at Amgen, said: “Bone complications like fracture, spinal cord compression and radiation or surgery to bone are devastating for multiple myeloma patients. Many of these patients suffer from renal impairment, which has limited their treatment options. XGEVA’s unique mechanism of action has the potential to prevent bone complications in multiple myeloma patients regardless of their renal status, fulfilling an important unmet medical need.”

Pfizer Presented New Data on XELJANZ® for Ulcerative Colitis at UEG Week 2016

Pfizer Igea Luca Dezzani

Pfizer Igea Luca Dezzani

October 15, 2016

Pfizer Inc. announced that three abstracts for XELJANZ® (tofacitinib citrate), being investigated in moderate to severe ulcerative colitis (UC), were presented at the United European Gastroenterology Week (UEG Week 2016), October 15-19 in Vienna, Austria. The tofacitinib presentations highlighted new research results from the Phase 3 Oral Clinical Trials for tofAcitinib in ulceratiVE colitis (OCTAVE) Induction trials, including one oral presentation looking at the impact of prior treatment with tumor necrosis factor inhibitors (TNFi) on efficacy endpoints. Furthermore, two abstracts were accepted as poster presentations, highlighting results by endoscopic response, and onset of action, respectively.

Michael Corbo, PhD, Chief Development Officer, Inflammation & Immunology, Pfizer Inc., said: “The new data to be presented at UEG Week deepen our understanding of the efficacy and safety profile of tofacitinib in ulcerative colitis. We know there is a significant unmet need in the UC community for additional treatment options and, if approved, tofacitinib may have the potential to offer patients and their physicians an oral treatment option that could address these unmet needs in the course of the disease.”

Tofacitinib is the first in a new class of medicines called Janus kinase (JAK) inhibitors under investigation for treating moderate to severe UC. Tofacitinib is a small molecule taken as a pill. It works on specific inflammatory responses thought to play a role in the inflammation connected with UC.

 

Treatment outcome data from The Prostate Cancer Registry presented for the first time at the ESMO Congress 2016

janssen-logo

Oct 10, 2016

Janssen-Cilag International NV has presented the first reported primary treatment outcome data from The Prostate Cancer Registry at the 2016 European Society for Medical Oncology (ESMO) Congress in Copenhagen, Denmark. The Prostate Cancer Registry is the first and largest prospective study of men with metastatic castration-resistant prostate cancer (mCRPC) in Europe. The preliminary data suggest that chemotherapy-naïve patients benefit more from treatment than post-chemotherapy patients. Furthermore, patients have a higher prostate-specific antigen (PSA) response when treated with androgen receptor-targeted agents than with taxanes, after first line docetaxel treatment.

“The Prostate Cancer Registry provides unique insights into the treatment of mCRPC patients from a large, real-world population. With enrolment now complete at over 3,000 patients across 16 countries, there is no other registry in advanced prostate cancer of this size that has produced such a large volume of data. The Prostate Cancer Registry is helping us to address a critical gap in our understanding of the real-world management of patients with mCRPC. This involves studying patients who have high rates of comorbidities and use multiple medications, who are usually excluded from clinical trials. Every patient is different and it is extremely important for clinicians to be able to understand how men with mCRPC respond to medications to ensure that we chose the very best treatment for each individual. I look forward to seeing further data as the Registry continues,” said Dr Simon Chowdhury, Guy’s Hospital, London.

U.S. FDA Accepts Biologics License Application for Romosozumab, Amgen and UCB Announce

amgen-ucb

Sept. 26, 2016

Amgen and UCB have announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for romosozumab. It is an investigational monoclonal antibody for treating osteoporosis in postmenopausal women at increased risk of fracture. Romosozumab works by binding and hindering the activity of the protein sclerostin, naturally occurring in the bone, thereby reducing bone resorption and increasing bone formation.

Sean E. Harper, M.D., executive vice president of Research and Development at Amgen, said: “We believe romosozumab could serve as an important therapeutic option for osteoporosis patients with an increased risk of fracture. We look forward to potentially providing a new therapy to address a critical unmet medical need and further supporting patients worldwide.”

The BLA was submitted on July 19, 2016. It was based on data from the pivotal Phase 3 placebo-controlled fracture study in postmenopausal women with osteoporosis (FRAME) in almost 7,200 patients.

“Once a patient suffers a fragility fracture, the burden of osteoporosis can have a tremendous impact on a patient’s life. We are pleased with the FDA’s acceptance of the BLA filing for review and hope to continue the regulatory process towards U.S. approval to introduce a potential therapy to reduce the risk of fractures for this patient population,” said Dr. Pascale Richetta, head of bone and executive vice president, UCB.

Two Cardiovascular Collaborations Announced by Amgen and Arrowhead Pharmaceuticals

amgen-arrowhead

Sept. 29, 2016

Amgen and Arrowhead Pharmaceuticals Inc. have announced two license and collaboration agreements to develop and commercialize RNA interference (RNAi) therapies for cardiovascular disease. Under the first agreement, Amgen receives a worldwide, exclusive license to Arrowhead’s novel, RNAi ARC-LPA program. The RNAi molecules are designed to decrease elevated lipoprotein, which is a genetically validated, independent risk factor for atherosclerotic cardiovascular disease. As per the second agreement, Amgen receives an option to a worldwide, exclusive license for a RNAi therapy for an undisclosed genetically validated cardiovascular target. Amgen will be wholly responsible for clinical development and commercialization in both agreements.

Sean E. Harper, M.D., executive vice president of Research and Development at Amgen, said: “Arrowhead’s expertise in RNAi makes them a valuable partner as we translate genetic discoveries into potential therapies that can improve health outcomes for patients. This collaboration builds upon our commitment to cardiovascular disease with targets that we believe are uniquely suited for RNAi-based therapy.”

“We have made great advances to our proprietary subcutaneous RNAi delivery vehicle and in RNAi trigger modification and stabilization that enable rapid development of new RNAi therapeutics across multiple disease areas. Our capabilities and platform technologies are becoming increasingly validated, so we feel that now is a great time to expand the reach of our technologies and partner with other companies to maximize the value of our assets. We are thrilled to be working with Amgen, one of the world’s leading biotechnology companies, on this collaboration. Amgen’s extensive development, regulatory, and commercial expertise makes them an ideal partner, and we look forward to a long and productive relationship,” said Christopher Anzalone, Ph.D., president and chief executive officer at Arrowhead.

Top 5 Causes of Drug Price Hike

Drug Price - Igea - Luca Dezzani

Over the years, the consistently rising medical expenditures has been affecting all the stakeholders of the current healthcare landscape around the globe. In fact, according to PricewaterhouseCoopers, the projected  growth rate of medical costs for the year 2017 will remain at 6.5%. Though the rate is the same as this year, it still outpaces the general economic inflation.

One of the top drivers of the skyrocketing health care costs is drug price hike. Given the fact that pharmaceutical companies play the major role in drug pricing, they have to consider all aspects to justify the value of their products. Through this,  they will be able to maintain profitability while ensuring the welfare of a larger patient population. The following will give us an overview of the most common factors that can significantly affect the price of drugs currently available in the market.

drug-price-hike

  1. Narrow competition in the marketplace

Due to a threat to profitability, particularly in the year 2009, many pharmaceutical companies ventured to merger and acquisitions while others decided to leave the industry. Hence, a decrease in the number of competitors gives large manufacturers a strong market power that is further influenced by the Food and Drug Administration’s (FDA’s) slow approval of new entrants into the pharmaceutical industry. This market structure leads to higher prices of drugs and other medical products.

  1. Research and development of new drugs

From idea to market, research and development (R&D) of new drugs entail complex processes. The whole course of R&D is not only long and tedious, but also enormously expensive. Most clinical research and trials involve recruitment of several patients and establishing multiple sites from different parts of the world. Thus, requiring huge budget.  Therefore, once it becomes approved, drug companies raise the price of their products to compensate for the high R&D costs and to sustain its supply in the market.

  1. Aging drugs and niche therapies

Due to very low profitability, some drugs that were approved years ago were dropped by manufacturers in exchange of newly discovered drugs with higher demand. Hence, shortages occur when some firms stop the production. Thus, leading to higher product price.

  1. Raw materials shortage

Raw materials shortage is one of the leading causes of drug shortage. In the marketplace, there may be several drug manufacturers, but few suppliers of raw materials. Multiple companies may import from other countries. However, the international supply chain may not always be reliable as it may face problems related to political issues, trade disputes, unfavorable climate conditions and contamination during transport.

  1. Drug shortage

Aside from raw materials shortage, multiple events may contribute to drug shortage. One of these is unanticipated demand, which occurs when the demand for a specific drug goes beyond the clinical demand predicted by pharmaceutical companies. This usually happens when a new indication of drug was discovered or in case of rapid progression of diseases. However, due to the strict rules governing the development and production of drugs, manufacturers cannot hasten the process to quickly top up the supply of their products in the market. Furthermore, another factor that may lead to drug shortage is manufacturing difficulties. From time to time, pharmaceutical companies may encounter problems related to the process of drug production such as outdated equipment, changes in the drug’s formulation, limited production capacity and quality control issues.

Amgen Announces Top-Line Results from Phase 3 KYPROLIS® (Carfilzomib) CLARION Study in Patients with Newly Diagnosed Multiple Myeloma

Amgen Igea Luca Dezzani

Amgen Igea Luca Dezzani

Sept. 27, 2016

Amgen has announced top-line results of the Phase 3 CLARION trial evaluating an investigational regimen of KYPROLIS® (carfilzomib), melphalan and prednisone (KMP) versus CLARION Study® (bortezomib), melphalan and prednisone (VMP) for 54 weeks in patients with newly diagnosed multiple myeloma who were not eligible for hematopoietic stem-cell transplant. The trial did not meet the primary endpoint of superiority in progression-free survival (PFS). The observed hazard ratio (KMP versus VMP) was 1.21 (95 percent CI, 0.90 – 1.64) while the data for overall survival, a secondary endpoint, are not yet mature. Neither result was statistically significant. These data will be submitted to a future medical conference and for publication.

Sean E. Harper, M.D., executive vice president of Research and Development at Amgen, said: “Based on studies in the KYPROLIS label, including the ENDEAVOR study, a head-to-head comparison of KYPROLIS to Velcade in patients with relapsed or refractory multiple myeloma, we know KYPROLIS to be a major advance in proteasome inhibitor therapy. The CLARION results, generated in the context of a melphalan-containing regimen, are disappointing, especially given the robust data we’ve seen in the second-line setting. However, the myeloma landscape has changed dramatically since the design of the CLARION study with very few newly diagnosed patients treated with melphalan-based regimens, particularly in the U.S.  We remain committed to exploring KYPROLIS in combination with other agents to advance the treatment of multiple myeloma.”