Discussions about costs are important. Too often patients struggle to afford the medicines they need. Our industry is committed to working with policymakers on solutions that enhance the competitive marketplace, lower costs for patients and promote continued medical innovation.

(OP-ED) How many times did you hear, "Here's to good health" this holiday season? As we age, we become increasingly aware of the importance of "good health." That's one reason so many people felt hopeful about a recent announcement by the Food and Drug Administration (FDA) that the agency approved more than 50 new molecular entities in 2018, including 16 approvals for cancer drugs, as well as drugs for HIV, Hepatitis C and rare diseases. Twenty of the new drugs came from companies with a footprint in New Jersey.

Newark-based BioNJ Member Rafael Pharmaceuticals, Inc. announced that the European Medicines Agency (EMA) has granted orphan drug designation to devimistat (CPI-613®), the Company's lead Altered Metabolism Directed (AMD) drug candidate, for the treatment of older patients (≥ 60 years) with relapsed or refractory Acute Myeloid Leukemia (AML). AML is a hematologic malignancy characterized by the accumulation of clonal myeloid progenitor cells ("blasts") in the blood or bone marrow. 

Cranbury-based BioNJ Member   Amicus Therapeutics  announced the initiation of a Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9 AAV9-CLN3 (AAV9-CLN3) gene therapy in children with CLN3 Batten disease. Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. 

Bridgewater-based BioNJ Member   Sanofi  announced that the  U.S. Food and Drug Administration  has approved the expanded use of Adacel (Tetanus Toxoid, Reduced Diphtheria Toxoid and Acellular Pertussis (Tdap) Vaccine Adsorbed) to include repeat vaccination to help protect against tetanus, diphtheria and pertussis. According to Sanofi, it is the first and only Tdap vaccine in the U.S. approved for a repeat dose in people 10 through 64 years of age, eight years or more after the first vaccination. 

The New England Journal of Medicine (NEJM) published positive results of the Phase 3 trial of Bridgewater-based BioNJ Member Sanofi's  Cablivi® (caplacizumab) in adults with acquired thrombotic thrombocytopenic purpura (aTTP). aTTP is a rare, life-threatening autoimmune blood disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), ischemia (restricted blood supply to parts of the body) and widespread organ damage, especially in the brain and heart.

Princeton-based BioNJ Member   Otsuka America, Inc. ,  and Click Therapeutics, Inc., announce that the companies have signed a collaboration agreement to develop and commercialize a prescription digital therapeutic for treatment of Major Depressive Disorder (MDD), with the intent to address unmet medical needs among this patient population and to improve outcomes. This collaboration will leverage Click's demonstrated ability to discover and validate a software application and deploy it commercially, with Otsuka's expertise in developing approved prescription therapies for patients with serious mental illnesses, including Otsuka's established development and commercialization capabilities. 

Parsippany-based BioNJ Member The Medicines Company announced that the 5th review of un-blinded safety and efficacy data by the Independent Data Monitoring Committee (IDMC) for inclisiran Phase 3 trials has been completed. The IDMC has once again recommended that the trials continue as designed and conducted, without modification.

Princeton-based BioNJ Member   Agile Therapeutics, Inc. announced that  it received final meeting minutes from its  December 11, 2018  meeting with the U.S. Food and Drug Administration's Division of Bone , Reproductive, and Urologic Products ("DBRUP"). The company met with DBRUP to discuss the design of a comparative wear study between Twirla ®  and Xulane ®  (the "comparative wear study") as suggested by FDA's  Office of New Drugs  ("OND") in its decision on the company's previously announced formal dispute resolution request. 

Princeton-based BioNJ Member   Bristol-Myers Squibb Company  announced that the European Commission has approved the combination of Opdivo  (nivolumab) 3 mg/kg plus Yervoy  (ipilimumab) 1 mg/kg ("low-dose") for the first-line treatment of patients with intermediate- and poor-risk advanced renal cell carcinoma (RCC). This decision represents the first approval of an Immuno-Oncology (I-O) combination therapy for patients with this type of cancer in the European Union.

Bridgewater-based BioNJ Member   Allergan plc  announced positive 3-month topline results from the second pivotal clinical trial of Bimatoprost SR, a first-in-class sustained-release, biodegradable implant for the reduction of IOP in patients with open-angle glaucoma or ocular hypertension. Positive results from the first Phase 3 trial were reported in  June 2018 . Bimatoprost SR is designed to lower IOP for at least 4 months, achieving clinical goals while freeing patients from daily eye drop regimens. 

Kenilworth-based BioNJ Member   Merck & Co. announced the first presentation of results from KEYNOTE-181, a Phase 3 trial investigating KEYTRUDA, Merck's anti-PD-1 therapy, as monotherapy for the second-line treatment of advanced or metastatic esophageal or esophagogastric junction carcinoma. In this pivotal study, KEYTRUDA met a primary endpoint by significantly improving overall survival (OS) in patients with squamous cell carcinoma or adenocarcinoma who progressed after standard therapy and whose tumors expressed PD-L1 (Combined Positive Score [CPS] ≥10), with a 31 percent reduction in the risk of death compared to chemotherapy (paclitaxel, docetaxel or irinotecan).

Kenilworth-based BioNJ Member   Merck announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Merck's anti-PD-1 therapy, for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC), based on the results of the Cancer Immunotherapy Trials Network (CITN)'s CITN-09/KEYNOTE-017 trial. 

Titusville-based BioNJ Member  The Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a Group Type II Variation Application to the European Medicines Agency (EMA) seeking approval of STELARA® (ustekinumab) for the treatment of adults with moderately to severely active ulcerative colitis (UC). Ustekinumab is a human monoclonal antibody that targets the interleukin (IL)-12 and IL-23 cytokines, which are believed to play an important role in the immune and inflammatory responses seen in immune-mediated diseases, such as UC and Crohn's disease.

Princeton-based BioNJ Member Genmab A/S announced that it has achieved a USD 75 million sales volume milestone in its DARZALEX ®(daratumumab) collaboration with BioNJ Member Janssen Biotech, Inc.  The milestone was triggered by confirmation by Janssen that sales of DARZALEX reached USD 2 billion in the calendar year of 2018. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize DARZALEX.  

Princeton -based Sandoz Inc., a division of BioNJ Member  Novartis, and Pear Therapeutics, Inc., announced the U.S. commercial launch of reSET-O(TM) for patients with Opioid Use Disorder (OUD). reSET-O, cleared by the U.S. Food and Drug Administration (FDA) in December, is immediately available. The reSET-O prescription digital therapeutic (PDT) is a 12-week cognitive behavioral therapy intended to be used in addition to outpatient treatment. 

Princeton -based Sandoz Inc., a division of BioNJ Member  Novartis, announced the US market introduction of SYMJEPI™ (epinephrine) 0.3 mg Injection for the emergency treatment of allergic reactions (Type 1), including anaphylaxis. Sandoz is launching this medicine as an affordable, single-dose, pre-filled syringe alternative to epinephrine auto-injectors. SYMJEPI will be rolled out through a phased launch, first in the institutional setting -- an established channel where Sandoz Inc. has significant experience and knowledge -- followed by introduction into the retail market.

Touted by East Hanover -based BioNJ Member  Novartis chief Vas Narasimhan as a potential blockbuster, the drugmaker's sickle cell disease (SCD) drug has secured the FDA's breakthrough therapy designation for its ability to prevent debilitatingly painful vaso-occlusive crisis for patients affected by the group of inherited red blood cell disorders that typically afflict those of African ancestry.

BioNJ Member Pfizer Inc., with offices in Madison, announced that the U.S. Food and Drug Administration (FDA) accepted for filing the company's New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Pfizer has submitted two NDAs based on two forms of tafamidis: meglumine salt and free acid. Tafamidis is the only product to complete a Phase 3 trial evaluating its efficacy, safety, and tolerability in patients with ATTR-CM, a rare, fatal and underdiagnosed condition.

BioNJ Member Pfizer Inc., with offices in Madison, announced the initiation of a Phase 2b/3 clinical trial for its oral JAK3 inhibitor, PF-06651600, for the treatment of patients with moderate to severe alopecia areata, a chronic autoimmune skin disease that causes hair loss on the scalp, face, or body and currently has no approved therapies.

Bridgewater-based BioNJ Member Eli Lilly and Company  announced a definitive agreement for Lilly to acquire  Loxo Oncology  for  $235.00  per share in cash, or approximately  $8.0 billion .  Loxo Oncology  is a biopharmaceutical company focused on the development and commercialization of highly selective medicines for patients with genomically defined cancers. The acquisition would be the largest and latest in a series of transactions Lilly has conducted to broaden its cancer treatment efforts with externally sourced opportunities for first-in-class and best-in-class therapies. 

Basking Ridge-based BioNJ Member  Ipsen announced that abobotulinumtoxinA (Dysport®) and its recombinant botulinum toxins pipeline are the subject of 50 posters at the 2019 TOXINS International Conference. Results are presented from basic science (in vivo, in vitro, ex vivo, in silico), to clinical (Phase I to IV) and patients and caregivers surveys. Data highlights include further differentiation of Dysport® in the treatment of spasticity and movement disorders, results from the first in-human study of a recombinant neurotoxin (rBoNT-E), real life data (ULIS-III) as well as a survey with insights from patients and caregivers on the burden of spasticity (Carenity).

Bedminster-based BioNJ Member Matinas BioPharma Holdings, Inc. announced they have signed an agreement with an undisclosed top global pharmaceutical company aimed to evaluate synergistic effects of Matinas' lipid-nano-crystal ("LNC") platform delivery technology with their partner's nucleic acid polymer technology. Formulations will be developed using Matinas' LNC delivery technology which enables the development of a wide range of difficult-to-deliver molecules.

Princeton-based BioNJ Member Evotec AG announced that its strategic alliance with BioNJ Member Celgene Corporation has reached another important scientific achievement, resulting in Celgene designating a programme and triggering a payment of $ 14.0 m to Evotec. The payment was received by year-end 2018. This scientific achievement was the advancement of a programme into the lead optimisation stage developed from Evotec's compound library using its induced pluripotent stem cell ("iPSC") based screening platform.

Bedminster-based  Aerie Pharmaceuticals, Inc.  announced the topline results of its pilot Phase 2 study of netarsudil ophthalmic solution in a Japanese-American population. The study was designed in accordance with the requirements of Japan's PMDA (Pharmaceuticals and  Medical Devices Agency ) to support the potential regulatory submission of netarsudil ophthalmic solution in  Japan . Netarsudil ophthalmic solution 0.02% is known by the name Rhopressa ®  in  the United States , where it is currently marketed.

ALK, with offices in Bedminster,  announced top-line results from a Phase III paediatric clinical trial of its sublingual allergy immunotherapy (SLIT) tablet for the treatment of ragweed-induced allergic rhinitis. The trial, which lasted three years and involved 1,022 participants in North America and Europe, was a Phase III, randomised, placebo-controlled trial to study the efficacy and safety of ALK's ragweed SLIT-tablet in children aged 5 to 17 with a history of ragweed-induced allergic rhinitis (also known as hay fever).

South Plainfield-based BioNJ Member  PTC Therapeutics, Inc.  announced the promotion of Dr. Mark Pykett  to Chief Scientific Officer and Dr. Neil Almstead  to Chief Technical Operations Officer. "Achievement of our long-term strategy requires accelerating our research engine with our small molecule and gene therapy platforms as well as fostering our manufacturing expertise to focus on supplying the needs of our advancing gene therapy franchise," said Stuart W. Peltz , Ph.D., Chief Executive Officer of  PTC Therapeutics .

Rutherford-based BioNJ Member  Cancer Genetics Inc.  announced it has appointed William Finger its new Executive Vice President, Precision Medicine and Biopharma Solutions, effective immediately. He  will be responsible for developing and overseeing the company's precision medicine and biopharma, and clinical trial business unit. Dr. Finger, who has more than 25 years of experience in the life sciences, diagnostics and lab services industry, most recently served as managing director, precision medicine and diagnostics for Kineticos. 

Warren-based  Aquestive Therapeutics Inc.  announced it has appointed Gary H. Slatko as Senior Vice President, Chief Medical Officer. Dr.  Slatko will be responsible for heading clinical and medical affairs for the company. He will report directly to CEO Keith J. Kendall. Prior to Aquestive, Dr. Slatko was a director in the Office of Medication Error Prevention and Risk Management at the U.S. Food and Drug Administration. Before that, he was Chief Medical Officer of ParagonRx. 

Bridgewater-based  Amneal Pharmaceuticals Inc.  announced it has named David A. Buchen its new Senior Vice President, Chief Legal Officer and Corporate Secretary. Mr.  Buchen, who will report directly to CEO and President Robert Stewart, will be responsible for leading the company's global legal, intellectual property and corporate compliance functions. Mr. Buchen, who has more than 25 years of experience in the pharmaceutical industry, most recently served as a consultant to various manufacturing, marketing and distribution companies. 

Navitas Life Sciences , a Princeton business of TAKE Solutions, has named a veteran pharmaceutical executive as its new Global Head of Clinical. Steve Galen, who has held leadership roles with companies including Covance, PRA, InVentiv Health and Syneos Health, will join the clinical, regulatory and safety solutions company's Princeton office. Mr. Galen has more than 20 years of pharma and contract research organization experience.

Vascular Therapies Inc. announced it has appointed a new CEO.  John McDermott, who has more than 30 years of experience in vascular technologies, will head the Cresskill-based company. Dr. Sriram Iyer, Founder of Vascular Therapies, has done an excellent job leading the company since inception and will continue as its Chief Scientific Officer," Bob Croce, Chairman of the Board of Directors of Vascular Therapies, Inc, said.

ALK, with offices in Bedminster,  announced the appointment of Christian G. Houghton as its new Head of Product Supply, which includes all global manufacturing activities. Mr. Houghton has been with ALK for 25 years and was previously Head of Chemistry, Manufacturing and Controls (CMC) and selected manufacturing sites.