New Drug Approvals - The Month That Was

(RTTNews) - The process of drug development is challenging. Some drugs pass the FDA muster easily, while some face a host of hurdles before getting the official stamp of approval.

The FDA approved 41 novel new drugs last year, compared to 27 in 2013, and perhaps the most in nearly 20 years. The number of new drug approvals from 2005 through 2014 has averaged nearly 27 per year.

As the first month of the New Year heads to a close, let's take a look at the drugs that crossed the regulatory finish line in January of 2015.

1. Savaysa - 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, and for the treatment of deep vein thrombosis, and pulmonary embolism.

Developed by Tokyo-based Daiichi Sankyo Co. Ltd., Savaysa was approved by the FDA on January 8, 2015.

Savaysa is an oral, once-daily drug that works by inhibiting factor Xa, which is important in the clotting of blood. In clinical trials, Savaysa was compared with the most commonly used oral anti-clotting drug - Warfarin - for its effects on rates of stroke and dangerous blood clots (systemic emboli).

Besides being as effective as Warfarin in preventing stroke-causing clots, Savaysa is associated with significantly less major bleeding compared to Warfarin, according to the FDA. The drug is expected to be commercially available in the U.S. in February 2015.

Savaysa is the fourth new oral anticoagulant (NOAC) to be approved by the FDA - the other three being - Boehringer Ingelheim's Pradaxa (approved on October 19th, 2010), Janssen's Xarelto (July 1st, 2011) and Bristol-Myers Squibb/Pfizer's Eliquis (December 28th, 2012).

Savaysa has been available in Japan since 2011 under the brand name Lixiana. In Europe, the drug is under regulatory review.

Like the other FDA-approved NOAC medications, Savaysa also sports a black-box warning, the strongest warning available for prescription drugs. Savaysa's black box label warning indicates that it should not be used in atrial fibrillation patients with a creatinine clearance greater than 95 milliliters per minute because of an increased risk of stroke compared with Warfarin.

The most serious risk with FDA-approved anti-clotting drugs is bleeding, including life-threatening bleeding.

While low-dose Vitamin K is recommended as an antidote in the event of bleeding injuries related to Warfarin, there are no reversal agents for the new oral anticoagulants.

Antidotes for the new oral anticoagulants are still under development - say Idarucizumab, which is being studied as a specific antidote for Pradaxa, and Andexanet alfa, which is being tested as an antidote for Xarelto, Eliquis and Pradaxa.

2. Rytary - for the treatment of Parkinson's disease, post-encephalitic parkinsonism, and parkinsonism that may follow carbon monoxide intoxication and / or manganese intoxication.

Developed by Impax Pharmaceuticals, a division of Impax Laboratories Inc. (IPXL), Rytary was approved by the FDA on January 8, 2015.

Rytary is an extended-release oral capsule formulation of carbidopa-levodopa, and should be swallowed whole or, for patients who have trouble swallowing, the capsule may be opened and the beads sprinkled on applesauce and consumed immediately. It should not be chewed, divided, or crushed.

The drug will be available for commercial distribution in 4 strengths - 23.75mg/95mg, 36.25mg/145mg, 48.75mg/195mg, and 61.25mg/245mg (carbidopa/levodopa) - in February 2015.

3. Cosentyx - to treat adults with moderate-to-severe plaque psoriasis. Developed by Novartis (NVS), Cosentyx was approved by the FDA on January 21, 2015.

Cosentyx, administered as an injection under the skin, works by inhibiting the action of IL-17A, a protein that is found in high concentrations in skin affected by psoriasis. Cosentyx is the first and only approved treatment targeting the IL-17 pathway.

In clinical trials, Cosentyx demonstrated superiority to Amgen/Pfizer's Enbrel, a current standard-of-care, in clearing psoriasis skin with a comparable safety profile. Enbrel works by blocking tumor necrosis factor alpha (TNF alpha), a protein that signals the body to create inflammation.

Cosentyx is approved with a Medication Guide, warning patients that the drug may increase the risk of getting an infection. The drug is already approved in the EU, Australia and Japan.

4. Natpara - to control hypocalcemia (low blood calcium levels) in patients with hypoparathyroidism.

Developed by *NPS Pharmaceuticals Inc. (NPSP), Natpara received FDA approval on January 23, 2015.

Natpara, a bioengineered replica of human parathyroid hormone, is administered by injection once daily, and it offers an alternative to patients whose calcium levels cannot be controlled on calcium supplementation and active forms of vitamin D.

In clinical trials, 42 per cent of Natpara-treated participants achieved normal blood calcium levels on reduced doses of calcium supplements and active forms of vitamin D, compared to 3 per cent of placebo-treated participants.

Natpara, which is designated as an orphan drug by the FDA, carries a boxed warning on the potential risk of bone cancer (osteosarcoma), and is only available through a restricted program under a Risk Evaluation and Mitigation Strategy. The drug is expected to be available in the U.S. in the second quarter of 2015.

*NPS Pharmaceuticals has agreed to be acquired by Shire plc (SHPG) for a total consideration of approximately $5.2 billion, and the transaction is anticipated to close in the first quarter of 2015.

5. Bexsero - to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroup B in individuals 10 years through 25 years of age.

Developed by Novartis (NVS), Bexsero is a vaccine that works by stimulating the body to produce its own antibodies against the meningococcal group B bacteria, a leading cause of bacterial meningitis. This vaccine was approved by the FDA on January 23, 2015.

Bexsero is already approved in the member states of the European Union, Australia and Canada for use in individuals from 2 months of age and older.

6. Triferic - for iron replacement and maintenance of hemoglobin in hemodialysis patients.

Developed by Rockwell Medical Inc. (RMTI), Triferic received FDA approval on January 26, 2015.

Triferic is delivered to hemodialysis patients via dialysate, and it works by replacing the ongoing iron losses that occur during their dialysis treatment.

In clinical trials, Triferic has demonstrated that it can effectively deliver sufficient iron to the bone marrow to maintain hemoglobin and not increase iron stores (ferritin). Triferic is the first product to be approved that can safely allow dialysis patients to maintain target hemoglobin without the need for IV iron.

If the FDA sticks to its deadline, the regulatory decision on the following new drug - as to whether or not it is approved - will be announced this month.

Raplixa - a dry powder topical formulation of fibrinogen and thrombin as supportive treatment where standard surgical techniques are insufficient for improvement of haemostasis in adults.

Developed by The Medicines Co. (MDCO), Raplixa is under FDA review, with a decision slated for January 31, 2015.

Raplixa has been recommended for approval by the Committee for Medicinal Products for Human Use in the European Union, and a final decision is awaited.

Stay tuned...