This column summarizes New Drug Applications (NDAs) for the third quarter 2014 (July through September 2014). In this quarter, FDA approved 38 NDAs.
Thirty (78.9%) NDAs were for small molecules while the remaining 8 molecules (21.1%) were large molecules. Similar to the previous quarter, there is a continued interest in working on the large molecules. In this quarter, 18 companies (47.4%) receiving NDA approvals were large companies and the remaining 20 companies (52.6%) were mid-sized and small companies. Ten drug molecules were granted the Orphan Drug Status (26.3%). Boehringer Ingelheim and Gilead Sciences bagged 3 NDAs each in this quarter. Tablets and injections were the preferred dosage forms. Four out of 38 (10.5%) NDAs were combination dosage forms, out of which Triumeq (indicated for HIV infection) had 3 different drugs.
FDA approved Ozurdex (dexamethasone) 0.7 mg, a sustained-release biodegradable steroid implant for the treatment of general diabetic macular edema (DME) patients. Ozurdex was originally approved in June 2014 as a treatment for DME in adult patients who have an artificial lens implant or who are scheduled for cataract surgery. Similarly, Minivelle (estradiol transdermal system) was initially approved by the FDA in October 2012 to treat moderate to severe vasomotor symptoms due to menopause. FDA approved a new indication with a new dose of Minivelle for the prevention of postmenopausal osteoporosis.
Tablets can be split correctly with the help of score marks. Acticlate tablets (150 mg) have been scored twice so that patients can take 3 doses of 50-mg doxycycline hyclate from a single tablet. FDA has approved Targiniq ER, a new extended-release pain reliever that contains a combination of oxycodone and naloxone. Targiniq ER is the second extended-release/long-acting opioid analgesic with FDAapproved labeling describing the product’s properties that are expected to reduce, but not totally prevent, abuse by snorting or injecting. FDA suggested Drug Safety Labeling Changes for Lumizyme which include a boxed warning, precautions, and adverse reactions.
Nine out of 12 (75%) novel new drugs (NNDs) were approved in the US before any other countries.
Eight out of the 38 (21.05%) NDAs received approval for a new formulation or a new indication or an altered patient population or a new dosage form for the same drug, etc. These molecules had received NDAs previously. New indications were approved for Imbruvica, Eylea, and Otezla. Spiriva Respimat was available as an inhalation powder.
A new formulation for an inhalation spray was approved this quarter. Similarly, Abilify Maintena is now available as a dual-chamber syringe. It was approved as a powder for suspension for injection. Beleodaq, Cerdelga, Keytruda, and Zydelig were among the novel new drugs for treating rare diseases. Zydelig is used to treat a total of 3 different types of cancers. Beleodaq and Zydelig received fast track approvals by FDA as these drugs have a potential to address unmet medical needs. Keytruda along with the Beleodaq and Zydelig got accelerated approvals in this quarter. Belsomra, Kerydin, Keytruda, Otezla, and Zydelig were First-in-Class drugs approved in this quarter. Breakthrough therapies are those where the drug with preliminary clinical evidence demonstrating that the drug may result in substantial improvement on at least 1 clinically significant endpoint over other available therapies. Keytruda and Zydelig were the 2 drugs considered as breakthrough therapies. FDA provides priority review (review in 6 months instead of the standard 10 months) to some drugs to potentially provide a significant advance in medical care. Beleodaq, Cerdelga, Keytruda, Orbactiv, and Zydelig obtained a priority review. Under the GAIN (Generating Antibiotics Incentives Now) Act, incentives are provided to bring new antibiotics to the market. Under this, Orbactiv was designated as a Qualified Infectious Disease Product (QIDP) by the authority of the GAIN Act.
As for naming trends, a trend of including a few letters from the scientific drug name or company name was observed, as noted in Invokamet (combination of Canagliflozin & Metformin) or Kabiven (Company name Fresenius Kabi).