Each year, CDER approves hundreds of new medications, most of which are variations of previously existing products, such as important new dosage forms of already-approved products, or cost-saving generic formulations. These new products contribute to quality of care, greater access to medication, more consumer choice, and a competitive marketplace that enhances affordability and public health. However, products in a small subset of these new approvals, that we refer to as novel new drugs, are among the more truly innovative products that often help advance clinical care to another level. At the end of each calendar year, CDER summarizes these new products.
Our annual summary reports the quantity of novel new drugs that FDA approved. However, fda also focus on thequality of many of these new drugs, their contributions to enhanced patient care, and the various regulatory tools CDER used to help ensure their safe and efficient development and approval.
This year’s field of novel new drugs will offer much to patients in need. FDA approved many new drugs to treat various forms of cancer, including four to treat multiple myeloma, and others to treat lung, skin, breast, brain, colorectal, and other cancers. This year’s field also includes new drugs to treat heart failure and high cholesterol, as well as the first approved reversal agent for a commonly-used blood thinner. FDA also approved new drugs for the treatment of patients with cystic fibrosis, and irritable bowel syndrome. In the area of infectious disease, our approvals include new treatments for urinary tract infections and chronic hepatitis C. For the second consecutive year, we approved more “orphan” drugs for rare diseases than any previous year in our history.
This work in effectively reviewing and approving new drugs is meaningful to the extent that we can also effectively ensure their safety. All of these newly approved products were required to meet our rigorous premarket safety standards — and they will all be part of a strong postmarket safety surveillance system watching how they perform after they are more widely used by larger patient populations. FDA will summarize our safety activities in a different report.
FDA hope this summary provides an appreciation of the expected impact that many of the novel new drug approvals of 2015 will have on patient care, as well as the valuable role CDER played in helping to bring these drugs to market.
CDER’s 2015 Novel New Drugs
45 novel new drugs
In calendar year 2015, FDA’s Center for Drug Evaluation and Research (CDER) approved 45 novel new drugs, approved as new molecular entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs).
Novel new drugs are often innovative products that serve previously unmet medical needs or otherwise significantly help to advance patient care and public health. NMEs have chemical structures that have never been approved before. However, in some cases an NME may have actions similar to earlier drugs and may not necessarily offer unique clinical advantages over existing therapies. This report summarizes all of the 2015 NME and novel new BLA approvals, emphasizing those that offer new and innovative treatments to patients in need.
The vertical bars in the graph below indicate the number of novel new drugs approved by CDER in each year of the past decade. CDER approved 45 novel new drugs in 2015. From 2006 through 2014, CDER has averaged about 28 novel new drug approvals per year.
Applications for new approvals remain steady
CDER approved a higher than average number of novel new drugs in 2015; however, the number of applications for these drugs that sponsors have submitted over time has remained relatively stable.
The points connected by lines in the graph below indicate the number of new NDA and BLA applications for new molecular entities and new therapeutic biologics CDER has received and filed for approval during the last 10 years. From 2006 through 2014, CDER filed an average of about 35 applications for novel new drugs per year. CDER estimates 40 filings for 2015, which is consistent with previous years in this decade.
Below lists CDER’s novel new drugs of 2015.* (see New Molecular Entity and Therapeutic Biologics of 2015 for their non-proprietary names, approval dates, and what they are used for.)
Addyi, Alecensa, Aristada, Avycaz, Bridion, Cholbam, Corlanor, Cosentyx, Cotellic, Cresemba, Daklinza, Darzalex, Empliciti, Entresto, Farydak, Genvoya, Ibrance, Kanuma, Kengreal, Kybella, Lenvima, Lonsurf, Natpara, Ninlaro, Nucala, Odomzo, Orkambi, Portrazza, Praluent, Praxbind, Repatha, Rexulti, Savaysa, Strensiq, Tagrisso, Tresiba, Unituxin, Uptravi, Varubi, Veltassa, Viberzi, Vraylar, Xuriden, Yondelis, Zurampic.
In 2015 CDER approved 45 novel new drugs. 45 novel new drug approvals in CY 2015 is more than the average number approved annually during the past decade. From 2006 through 2014 CDER averaged about 28 novel new drug approvals per year.
Impact on Public Health
Many of the 45 novel new drugs CDER approved in 2015 are notable for their potential positive impact and unique contributions to quality medical care and public health.
First-in-Class
CDER identified 16 of the 45 novel new drugs approved in 2015 (36%) as First-in-Class, one indicator of the innovative nature of a drug. These drugs often have mechanisms of action different from those of existing therapies. This First-in-Class approval rate is one factor that suggests the 2015 group of novel new approvals is a field comprised of many innovative products.
Addyi, Bridion, Corlanor, Cosentyx, Darzalex, Empliciti, Entresto, Ibrance, Kanuma, Nucala, Orkambi, Praluent, Praxbind, Strensiq, Unituxin, Xuriden
Noteworthy First-in-Class products include:
Bridion – To reverse post-surgical neuromuscular blockade caused by certain kinds of anesthesia
Ibrance – To treat advanced (metastatic) breast cancer
Praxbind – To reverse adverse anticoagulant effects caused by the blood-thinner drug, dabigatran
Ibrance – To treat advanced (metastatic) breast cancer
Praxbind – To reverse adverse anticoagulant effects caused by the blood-thinner drug, dabigatran
Drugs for Rare Diseases
About 47% of the novel new drugs approved in 2015 (21 of 45) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans. This is significant because patients with rare diseases often have few or no drugs available to treat their conditions.
Alecensa, Cholbam, Cotellic, Cresemba, Darzalex, Empliciti, Farydak, Kanuma, Lenvima, Natpara, Ninlaro, Orkambi, Portrazza, Praxbind, Repatha*, Strensiq, Tagrisso, Unituxin, Uptravi, Xuriden, Yondelis
* Repatha was submitted with two indications. One indication received Orphan designation while the other did not.
Noteworthy examples of drugs to treat rare diseases among the 2015 novel new drugs include:
Kanuma – To treat lysosomal acid lipase deficiency, a rare inherited genetic disorder that does not allow the body to produce an enzyme responsible for breaking down fats, and can lead to liver disease, cardiovascular disease, and life-threatening organ damage.
Orkambi – A therapy for the lung disease, cystic fibrosis.
Strensiq – Long-term enzyme replacement therapy in patients with infantile- and juvenile – onset hypophosphatasia, a serious and sometimes fatal bone disease.
Unituxin – To treat pediatric patients with high-risk neuroblastoma (brain tumors).
Xuriden – To treat patients with hereditary orotic aciduria, a condition that can result in blood abnormalities (anemia, decreased white blood cell count, decreased neutrophil count), urinary tract obstruction, failure to thrive, and developmental delays.
Orkambi – A therapy for the lung disease, cystic fibrosis.
Strensiq – Long-term enzyme replacement therapy in patients with infantile- and juvenile – onset hypophosphatasia, a serious and sometimes fatal bone disease.
Unituxin – To treat pediatric patients with high-risk neuroblastoma (brain tumors).
Xuriden – To treat patients with hereditary orotic aciduria, a condition that can result in blood abnormalities (anemia, decreased white blood cell count, decreased neutrophil count), urinary tract obstruction, failure to thrive, and developmental delays.
Notable Novel New Drugs of 2015: Another strong year for quality
In addition to the noteworthy examples of innovative First-in-Class and “orphan” new products mentioned previously above, the 2015 novel new drug field also includes a variety of other notable drugs. These include the antibacterial drug Avycaz, to treat complicated intra-abdominal infections and complicated urinary tract infections, and the antifungal product Cresemba, to treat invasive aspergillosis and invasive mucormycosis, rare but serious infections. Also, the heart drugs, Entresto, to treat heart failure, and Corlanor, to reduce hospitalization from worsening heart failure; and the hypercholesterolemia (high cholesterol) treatments, Praluent, to treat certain patients with hard to treat heterozygous familiar hypercholesterolemia and Repatha, to treat this same condition as well as homozygous familial hypercholesterolemia (a rare disease).
Additional noteworthy cancer treatments include Darzalex, Empliciti, Farydak, and Ninlaro, to treat patients with multiple myeloma (a type of bone cancer), Alecensa and Tagrisso, to treat certain patients with non-small cell lung cancer, Cotellic, to treat certain patients with metastatic melanoma (skin cancer), Lonsurf, for the treatment of certain patients with metastatic colorectal cancer, and, Yondelis,for treatment of soft tissue carcinoma (cancer of the inner or outer surfaces of the body).
The year’s notable approvals also include Viberzi, to treat patients who have irritable bowel syndrome with diarrhea (IBS-D), Veltassa, to treat hyperkalemia (elevated potassium in the blood), and Daklinza, to treat chronic hepatitis C virus genotype 3 infections.
INNOVATION
Methods for expediting innovative novel new drugs to market
CDER used a number of regulatory methods to expedite the development and approval of novel new drugs in 2015. These involved the following four expedited pathways: Fast Track, Breakthrough, Priority Review, and Accelerated Approval.
Fast Track
Fourteen of the 2015 novel new drugs (31%) were designated by CDER as Fast Track, meaning drugs with the potential to address unmet medical needs. Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to drug developers and by enabling CDER to review portions of a drug application ahead of the submission of the complete application.
Avycaz, Corlanor, Cotellic, Daklinza, Darzalex, Entresto, Genvoya, Kanuma, Lonsurf, Orkambi, Portrazza, Strensiq, Tagrisso, Viberzi
Breakthrough
CDER designated ten of the 2015 novel new drugs (22%) as Breakthrough therapies, meaning drugs with preliminary clinical evidence demonstrating that the drug may result in substantial improvement on at least one clinically significant endpoint (i.e., study result) over other available therapies. A breakthrough therapy designation includes all of the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. Breakthrough status is designed to help shorten the development time of a potential new therapy.
Alecensa, Darzalex, Empliciti, Ibrance, Kanuma, Orkambi, Praxbind, Strensiq, Tagrisso, Xuriden
Priority Review
Twenty-four of the 2015 novel new drugs (53%) were designated Priority Review, in which CDER determined that the drug could potentially provide a significant advance in medical care and set a target to review the drug within six months instead of the standard 10 months.*
Alecensa, Avycaz, Bridion, Cholbam, Corlanor, Cotellic, Cresemba, Daklinza, Darzalex, Empliciti, Entresto, Farydak, Ibrance, Kanuma, Lenvima, Ninlaro, Orkambi, Praxbind, Strensiq, Tagrisso, Unituxin, Viberzi, Xuriden, Yondelis
*In some instances, priority review is assigned as a result of the sponsor redeeming a voucher for priority review under CDER’s Priority Review Voucher program, which may mean the drug does not potentially provide a significant advance. Such drugs are not included in the list above.
Accelerated Approval
CDER approved six of the 2015 novel new drugs (13%) under FDA’s Accelerated Approval program, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments. This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that we consider reasonably likely to predict a clinical benefit of the drug. Once Accelerated Approval is granted, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug to patients who need it.
Alecensa, Darzalex, Farydak, Ibrance, Tagrisso, Praxbind
Overall use of expedited development and review methods
Twenty-seven of the 2015 novel new drugs (60%) were designated in one or more expedited categories of Fast Track, Breakthrough, Priority Review, and/or Accelerated Approval. Each of these designations helps expedite the speed of the development and/or approval process and is designed to help bring important medications to the market as quickly as possible.
Alecensa, Avycaz, Bridion, Cholbam, Corlanor, Cotellic, Cresemba, Daklinza, Darzalex, Empliciti, Entresto, Farydak, Genvoya, Ibrance, Kanuma, Lenvima, Lonsurf, Ninlaro, Orkambi, Portrazza, Praxbind, Strensiq, Tagrisso, Unituxin, Viberzi, Xuriden, Yondelis
Qualified Infectious Disease Program Designations
The Generating Antibiotics Incentives Now Act (GAIN Act) provides incentives to help bring new antibiotics and other antimicrobials to market. A drug with particular promise can be designated as a Qualified Infectious Disease Product (QIDP) by authority of the GAIN Act. In 2015, CDER approved two novel new drugs with this designation.
| NO. | DRUG NAME | ACTIVE INGREDIENT | DATE | FDA-APPROVED USE ON APPROVAL DATE |
|---|---|---|---|---|
| 45. | Zurampic | lesinurad | 12/22/2015 | To treat high blood uric acid levels associated with gout |
| 44. | Uptravi | selexipag | 12/22/2015 | To treat pulmonary arterial hypertension |
| 43. | Bridion | sugammadex | 12/15/2015 | To reverse effects of neuromuscular blocking drugs used during surgery |
| 42. | Alecensa | alectinib | 12/11/2015 | To treat ALK-positive lung cancer |
| 41. | Kanuma | sebelipase alfa | 12/8/2015 | To treat patients with a rare disease known as lysosomal acid lipase (LAL) deficiency |
| 40. | Empliciti | elotuzumab | 11/30/2015 | To treat people with multiple myeloma who have received one to three prior medications |
| 39. | Portrazza | necitumumab | 11/24/2015 | To treat patients with advanced (metastatic) squamous non-small cell lung cancer (NSCLC) who have not previously received medication specifically for treating their advanced lung cancer Press Release |
| 38. | Ninlaro | ixazomib | 11/20/2015 | To treat people with multiple myeloma who have received at least one prior therapy |
| 37. | Darzalex | daratumumab | 11/16/2015 | To treat patients with multiple myeloma who have received at least three prior treatments. Press Release |
| 36. | Tagrisso | osimertinib | 11/13/2015 | To treat certain patients with non-small cell lung cancer Press Release |
| 35. | Cotellic | cobimetinib | 11/10/2015 | To be used in combination with vemurafenib to treat advanced melanoma that has spread to other parts of the body or can’t be removed by surgery, and that has a certain type of abnormal gene (BRAF V600E or V600K mutation) Press Release Drug Trials Snapshot |
| 34. | Genvoya | a fixed-dose combination tablet containing elvitegravir, cobicistat, emtricitabine, and tenofovir alafenamide | 11/5/2015 | For use as a complete regimen for the treatment of HIV-1 infection in adults and pediatric patients 12 years of age and older Press Release Drug Trials Snapshot |
| 33. | Nucala | mepolizumab | 11/4/2015 | For use with other asthma medicines for the maintenance treatment of asthma in patients age 12 years and older. Press Release |
| 32. | Strensiq | asfotase alfa | 10/23/2015 | To treat perinatal, infantile and juvenile-onset hypophosphatasia (HPP). Press Release Drug Trials Snapshot |
| 31. | Yondelis | trabectedin | 10/23/2015 | To treat specific soft tissue sarcomas (STS) – liposarcoma and leiomyosarcoma – that cannot be removed by surgery (unresectable) or is advanced (metastatic). Press Release Drug Trials Snapshot |
| 30. | Veltassa | patiromer for oral suspension | 10/21/2015 | To treat hyperkalemia, a serious condition in which the amount of potassium in the blood is too high. Press Release Drug Trials Snapshot |
| 29. | Praxbind | idarucizumab | 10/16/2015 | For use in patients who are taking the anticoagulant Pradaxa (dabigatran) during emergency situations when there is a need to reverse Pradaxa’s blood-thinning effects. Press Release Drug Trials Snapshot |
| 28. | Aristada | aripiprazole lauroxil | 10/6/2015 | To treat adults with schizophrenia Press Release Drug Trials Snapshot |
| 27. | Tresiba | insulin degludec injection | 9/25/2015 | To improve blood sugar (glucose) control in adults with diabetes mellitus Press Release Drug Trials Snapshot |
| 26. | Lonsurf | trifluridine and tipiracil | 9/22/2015 | To treat patients with an advanced form of colorectal cancer who are no longer responding to other therapies Press Release Drug Trials Snapshot |
| 25. | Vraylar | cariprazine | 9/17/2015 | To treat schizophrenia and bipolar disorder in adults Press Release Drug Trials Snapshot |
| 24. | Xuriden | uridine triacetate | 9/4/2015 | To treat patients with hereditary orotic aciduria Press Release Drug Trials Snapshot |
| 23. | Varubi | rolapitant | 9/2/2015 | To prevent delayed phase chemotherapy-induced nausea and vomiting (emesis) Press Release Drug Trials Snapshot |
| 22. | Repatha | evolocumab | 8/27/2015 | To treat certain patients with high cholesterol Press Release Drug Trials Snapshot |
| 21. | Addyi | flibanserin | 8/18/2015 | To treat acquired, generalized hypoactive sexual desire disorder (HSDD) in premenopausal women Press Release Drug Trials Snapshot |
| 20. | Daklinza | daclatasvir | 7/24/2015 | To treat chronic hepatitis C virus (HCV) genotype 3 infections Press Release Drug Trials Snapshot |
| 19. | Odomzo | sonidegib | 7/24/2015 | To treat patients with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, or who are not candidates for surgery or radiation therapy. Press Release Drug Trials Snapshot |
| 18. | Praluent | alirocumab | 7/24/2015 | To treat certain patients with high cholesterol Press Release Drug Trials Snapshot |
| 17. | Rexulti | brexpiprazole | 7/10/2015 | To treat schizophrenia and as an add on to an antidepressant to treat major depressive disorder Press Release Drug Trials Snapshot (treatment of major depressive disorder) Drug Trials Snapshot (treatment of schizophrenia) |
| 16. | Entresto | sacubitril/valsartan | 7/7/2015 | To treat heart failure Press Release Drug Trials Snapshot |
| 15. | Orkambi | lumacaftor 200 mg/ivacaftor 125 mg | 7/2/2015 | To treat cystic fibrosis Press Release Drug Trials Snapshot |
| 14. | Kengreal | canegrelor | 6/22/2015 | To prevent the formation of harmful blood clots in the coronary arteries for adult patients undergoing percutaneous coronary intervention Press Release Drug Trials Snapshot |
| 13. | Viberzi | eluxadoline | 5/27/2015 | To treat irritable bowel syndrome with diarrhea (IBS-D) in adult men and women. Press Release Drug Trials Snapshot |
| 12. | Kybella | deoxycholic acid | 4/29/2015 | To treat adults with moderate-to-severe fat below the chin, known as submental fat Press Release Drug Trials Snapshot |
| 11. | Corlanor | ivabradine | 4/15/2015 | To reduce hospitalization from worsening heart failure. Press Release Drug Trials Snapshot |
| 10. | Cholbam | cholic acid | 3/17/2015 | To treat pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders Press Release Drug Trials Snapshot (bile acid synthesis disorders) Drug Trials Snapshot (peroxisomal disorders) |
| 9. | Unituxin | dinutuximab | 3/10/2015 | To treat pediatric patients with high-risk neuroblastoma Press Release Drug Trials Snapshot |
| 8. | Cresemba capsuleinjection | isavuconazonium sulfate | 3/6/2015 | To treat adults with invasive aspergillosis and invasive mucormycosis, rare but serious infections Press Release Drug Trials Snapshot (aspergillosis) Drug Trials Snapshot (mucormycosis) |
| 7. | Avycaz | ceftazidime-avibactam | 2/25/2015 | To treat adults with complicated intra-abdominal infections (cIAI), in combination with metronidazole, and complicated urinary tract infections (cUTI), including kidney infections (pyelonephritis), who have limited or no alternative treatment options. Press Release Drug Trials Snaphot (cIAI) Drug Trials Snapshot (cUTI) |
| 6. | Farydak | panobinostat | 2/23/2015 | To treat patients with multiple myeloma Press Release Drug Trials Snapshot |
| 5. | Lenvima | lenvatinib | 2/13/2015 | To treat patients with progressive, differentiated thyroid cancer (DTC) whose disease progressed despite receiving radioactive iodine therapy (radioactive iodine refractory disease). Press Release Drug Trials Snapshot |
| 4. | Ibrance | palbociclib | 2/3/2015 | To treat advanced (metastatic) breast cancer Press Release Drug Trials Snapshot |
| 3. | Natpara | parathyroid horomone | 1/23/2015 | To control hypocalcemia (low blood calcium levels) in patients with hypoparathyroidism Press Release Drug Trials Snapshot |
| 2. | Cosentyx | secukinumab | 1/21/2015 | To treat adults with moderate-to-severe plaque psoriasis Press Release Drug Trials Snapshot |
| 1. | Savaysa | edoxaban | 1/8/2015 | To reduce the risk of stroke and dangerous blood clots (systemic embolism) in patients with atrial fibrillation that is not caused by a heart valve problem Press Release Drug Trials Snapshot (atrial fibrillation) Drug Trials Snapshot (venous thromboembolism) |
More important than the quantity of novel new drugs approved by CDER in 2015 is their quality and the important new roles they are serving to advance medical care.
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