Skip links

On the Rise: 2016 505(b)(2) NDA Approvals

On the Rise: 2016 505(b)(2) NDA Approvals

The total number of novel drug approvals in 2016 decreased by approximately half from last year (22 in 2016, from 45 in 2015) and is below the 10-year average of 29 drug approvals per year. Of these, how many were 505(b)(2)s? Were the number of 505(b)(2) approvals in 2016 similarly decreased or on par with previous years? A search of Camargo’s proprietary database provides informative results.

Novel Drug Approvals (NMEs)

In 2016, 22 novel drugs (new molecular entities or novel biologics) were approved, despite similar numbers of applications submitted for these products as in recent years (see also, 2015 505(b)(2) NDA approvals blog posting).

There is significant clinical value in the drugs that were approved, including the first treatment for patients with spinal muscular atrophy (Spinraza™), the first drug approved to treat Duchenne muscular dystrophy (Exondys 51™), a new drug to treat hallucinations and delusions in people with Parkinson’s disease (Nuplazid™), two new treatments for patients with hepatitis C (Zepatier™ and Epclusa®), and a treatment for patients with a rare chronic liver disease – primary biliary cirrhosis (Ocaliva). New oncology drugs for ovarian cancer, bladder cancer, soft tissue sarcoma, and chronic lymphocytic leukemia were also approved in 2016, as were two novel imaging agents to detect certain tumors.

The following statistics on these new drug approvals, as listed in CDER’s Novel Drugs Summary, were noted:

  • 9 (41%) of the 22 novel drug approvals were for rare or “orphan” diseases that affect 200,000 or fewer individuals in the US.
  • 15 (68%) were awarded priority review (6 month review time instead of 10 months).
  • Expedited approval pathways (Fast Track, Breakthrough, Priority Review, and Accelerated approval pathways) were used frequently (16 [73%] of drugs approved in 2016 took advantage one or more of these pathways) to speed up the approval process and bring important medications to market as quickly as possible.
  • 21 (95%) of 22 novel drugs of 2016 were approved in the “first cycle” of review (all except Xiidra™ for the treatment of dry eye disease, which received a complete response letter).
  • 19 (85%) of the 22 of the novel drugs approved in 2016 were approved in the US prior to receipt of approval in any other country.

505(b)(2) Approvals

Using Camargo’s proprietary database, we have performed a similar analysis of 2016 approvals that used the 505(b)(2) regulatory pathway. Unlike the novel drug approvals, which decreased dramatically from 2015, the number of 505(b)(2) drug approvals in 2016 increased, reaching a 13-year high of 48 (up from 2015’s 44).

Number of 505(b)(2) approvals each year from 2004 - 2016
*Number confirmed to date; however, there are some approval letters that cite 505(b) only and review documents are not yet available to confirm the regulatory approval pathway
Source: Camargo proprietary database


Of the novel drug approvals (22), 2 NMEs (9%) were approved by the 505(b)(2) pathway. This is similar to 2015, in which 4 (9%) of 45 novel drugs were approved as 505(b)(2)s.

The 2 505(b)(2)s approved as NMEs are:

  • Epclusa (sofosbuvir and velpatasvir) tablet 400 mg/100 mg (Gilead Sciences Inc)
    • Approved by the Office of Antimicrobial Products for the treatment of adult patients with chronic hepatitis C virus (HCV) genotypes 1, 2, 3, 4, 5, or 6 infection:
      • without cirrhosis or with compensated cirrhosis
      • with decompensated cirrhosis for use in combination with ribavirin
    • Granted Priority Review and Breakthrough Therapy designation
  • Netspot (gallium dotatate GA-68; Advanced Accelerator Applications USA Inc)
    • Approved by the Office of Drug Evaluation IV; Netspot, after radiolabeling with Ga-68, is a radioactive diagnostic agent indicated for use with positron emission tomography (PET) for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult and pediatric patients.
    • Granted Priority Review and Orphan Drug Designation

Upon approval, each NDA is assigned a chemical type, such as “New Molecular Entity” (Type 1) or “drug already marketed without approval” (Type 7) to characterize the newness of the drug product. The top categories are similar to 2015: New Manufacturer (Type 7; 47%), New Dosage Form (Type 3; 19%), New Combination (Type 4; 11%), and Drug Already Marketed without Approval (Type 7; 11%). As shown below, one drug was approved with two chemical types (New Active Ingredient and New Combination). One of the drugs approved as Type 7 (drug marketed without approval) was a literature-only NDA (Akovaz; NDA208289) (see Camargo’s recent blog post on how to get an NDA approved with literature alone). Also, as discussed above, 2 approvals were classified as NMEs, supporting the assertion that sponsors can get NMEs, with the associated exclusivity, approved through the 505(b)(2) regulatory pathway.

505(b)(2)s by Chemical Type


Join the Crowd

There is a reason why more people are doing more 505(b)(2).

Camargo is the industry leader in 505(b)(2), and has contributed to 1 in 5 recent 505(b)(2) NDA approvals. Contact us to learn more about how we can help ensure the most time efficient and cost-effective development path to NDA approval.