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.

With yet another month of 2015 coming to a close, let's take a look at the drugs that crossed the regulatory finish line in March.

1. Elepsia XR - indicated for adjunctive therapy in the treatment of partial onset seizures in patients 12 years of age and older with epilepsy was approved by the FDA on March 2, 2015.

Developed by Sun Pharma Advanced Research Co.Ltd., Elepsia XR, prescribed as once a day tablet, is available in two strengths - 1000 mg and 1500 mg. The active ingredient in Elepsia XR is Levetiracetam, which is said to work by slowing abnormal nerve impulses in the brain.

Business intelligence provider GBI Research estimates that the epilepsy therapeutics market value in the eight major countries — the US, Canada, France, Germany, Italy, Spain, the UK and Japan — will increase from $3.4 billion in 2012 to $4.5 billion by 2019, at a modest Compound Annual Growth Rate (CAGR) of 3.9%.

UCB's Keppra, GlaxoSmithKline's Lamictal and Trobalt/Potiga, Pfizer's Dilantin, Abbott's Depakote, and Novartis' Tegretol and Trileptal, and Eisai's Fycompa are some of the other approved epilepsy drugs.

Analysts expect Elepsia XR's peak annual sales to be around $50 million.

2. Opdivo injection - for the treatment of patients with metastatic squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy was approved by the FDA on March 4, 2015, more than three months ahead of the decision date of June 22, 2015. This approval marks the second indication for the drug.

Developed by Bristol-Myers Squibb (BMY), Opdivo works by inhibiting the cellular pathway known as PD-1 protein on cells that blocks the body's immune system from attacking cancerous cells.

In clinical trials, on average, participants who received Opdivo lived 3.2 months longer than those participants who received Docetaxel, an anticancer agent commonly used in the second-line treatment of NSCLC.

NSCLC is the most common type of lung cancer. The value of NSCLC market in the eight major countries is expected to increase from $5.1 billion in 2013 to $7.9 billion in 2020, according to GBI Research.

Roche's Avastin and Tarceva, Eli Lilly's Alimta and Gemzar, and Sanofi's Taxotere are some of the commonly used treatments in lung cancer.

The FDA previously approved Opdivo to treat patients with unresectable (cannot be removed by surgery) or metastatic melanoma who no longer respond to other drugs under the regulatory agency's accelerated approval program.

Some analysts project peak sales of Opdivo to be as high as $10 billion.

The most common side effects of Opdivo are fatigue, shortness of breath, musculoskeletal pain, decreased appetite, cough, nausea and constipation.

3. Zarxio - is the first biosimilar to be greenlighted in the U.S., following its approval by the FDA on March 6, 2015, under 351(k) regulatory pathway.

Developed by Sandoz Inc., the generic pharmaceuticals division of Novartis (NVS), Zarxio is biosimilar to Amgen Inc.'s (AMGN) Neupogen, which was originally licensed in 1991. Neupogen raked in sales of $1.2 billion in 2014.

Zarxio is approved for the same indications as Neupogen, namely, for patients with cancer receiving myelosuppressive chemotherapy; patients with acute myeloid leukemia receiving induction or consolidation chemotherapy; patients with cancer undergoing bone marrow transplantation; patients undergoing autologous peripheral blood progenitor cell collection and therapy; and patients with severe chronic neutropenia.

The active ingredient in Amgen's Neupogen is filgrastim and that in Zarxio is known as filgrastim-sndz. The same filgrastim product of Sandoz is currently on the market in Europe and in more than 40 countries outside the US, under the brand name Zarzio.

Amgen and Sandoz are embroiled in a patent suit over the biosimilar - with Amgen even requesting for a preliminary injunction to stop Sandoz from launching Zarxio in the U.S. However, the verdict delivered by the US District Court for North California on March 19, 2015 has gone in favor of Sandoz - with the Court denying Amgen's request. An appeal was filed by Amgen in the US Court of Appeals for the Federal Circuit on March 25, 2015.

Zarxio is not likely to be launched in the U.S. before May 11, 2015, or until the Federal Circuit's ruling on Amgen's appeal, whichever is earlier, according to reports.

4. Cresemba - a new antifungal drug to treat adults with invasive aspergillosis and invasive mucormycosis was approved by the FDA on March 6, 2015.

Developed by Astellas in partnership with Basilea Pharmaceutica International Ltd., Cresemba belongs to a class of drugs called azole antifungal agents, which target the cell membrane of a fungus. The drug, whose active ingredient is isavuconazonium sulfate, is available in oral and intravenous formulations.

The most common side effects associated with Cresemba include nausea, vomiting, diarrhea, headache, back pain and shortness of breath.

Cresemba is under review in the EU, and a decision is expected in the fourth quarter of 2015.

5. Unituxin - was approved as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a type of cancer that most often occurs in young children, by the FDA on March 10, 2015.

Unituxin is the first immunotherapy drug for children with cancer, and is only the third drug that has received initial FDA approval for a pediatric cancer in over 20 years.

Developed by United Therapeutics Corp. (UTHR), Unituxin is a monoclonal antibody that works by targeting tumor-associated antigens located on the surfaces of cancer cells to activate a patient's immune system against the cancer cells.

Unituxin irritates nerve cells, causing severe pain that requires treatment with intravenous narcotics and can also cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly following completion of the infusion. The drug sports a Boxed Warning alerting patients and health care professionals about the serious side effects associated with its use.

6. Cholbam - as a once-daily treatment for cholic acid deficiency in bile acid synthesis disorders due to single enzyme defects was approved by the FDA on March 17, 2015.

Developed by Asklepion Pharmaceuticals LLC, Cholbam is the first FDA approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders)

Retrophin (RTRX) will acquire worldwide rights to Cholbam under an agreement that was signed with Asklepion early this year. Cholbam will have seven years market exclusivity in the United States conferred by its designation as an orphan drug.

Bile acid synthesis disorders caused by single enzyme defects prevent the liver from properly producing bile acids that help the intestines absorb essential fats, vitamins and nutrients. The genetically damaged pathway instead produces toxic products that damage the liver.

Cholbam, whose active ingredient is cholic acid, works by down-regulating cholesterol 7alpha-hydroxylase, the rate limiting step in bile acid synthesis. In doing so, it inhibits the production and accumulation of hepatotoxic and cholestatic bile acid precursors.

Want to know which drugs are facing FDA decision this month?

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