Discovery Laboratories was boosted by the news that the U.S. FDA finally approved its Surfaxin (lucinactant) for the prevention of respiratory distress syndrome (RDS) in premature infants with high risk of the breathing disorder.

The US specialty pharma firm has previously failed on four occasions to win the regulators nod. The company had received a complete response letter nearly two years from the U.S. agency noting its concerns on “certain aspects of a Surfaxin biological activity test” that should be addressed before the drug could be given the green light.

Respiratory distress syndrome (RDS) is a potentially fatal breathing disorder that affects premature babies who are born with an insufficient amount of pulmonary surfactant, a substance produced naturally in the lungs and essential for breathing. It is estimated that around 90,000 premature infants in the USA are treated annually with currently available animal-derived surfactants along with mechanical ventilation to help them breathing.

Discovery Labs’ Surfaxin is the fifth agent cleared by FDA to treat RDS in premature infants. The other approved surfactants include Survanta (beractant), Curosurf (poractant alpha), Infasurf (calfactant), and Exosurf (colfosceril palmitate). Discovery Labs hopes its Surfaxin, as the first approved synthetic, peptide-containing surfactant, can replace all these conventional medicines in preventing RDS.

Results from a multinational phase 3 clinical trial has demonstrated the safety and efficacy of Surfaxin for the prevention of RDS in premature infants. In the late-stage study, nearly 1,300 premature infants received either Surfaxin, the no-longer marketed Exosurf or Abbott Laboratories’ Survanta within 30 minutes of birth. Surfaxin demonstrated significant improvement in both RDS at 24 hours after birth and RDS-related mortality through two weeks, when compared with Exosurf, which was marketed by GSK previously.

Discovery chief executive Thomas Amick was very optimistic on the approval of Surfaxin. He said the approval of Surfaxin is “an important medical advancement for the neonatology community and parents of preterm infants who will soon have an effective alternative to animal-derived surfactants to prevent the development of RDS”.

Discovery Labs said the company anticipates that SURFAXIN will be commercially available in the U.S. late this year.

The European Commission has granted AstraZeneca the right to market Caprelsa (vandetanib) as a treatment of aggressive and symptomatic medullary thyroid cancer (MTC) in patients with unresectable locally advanced or metastatic disease.

The British pharmaceutical company said Caprelsa is the first approved treatment for advanced MTC in Europe. The approval is expected as the oral kinase inhibitor received positive opinion from the European Medicines Agency for the indication last November.

CHMP, an advisory committee to the European agency, made the positive recommendation for Caprelsa based the data from late stage studies of the drug, which included ZETA study, a double-blind trial of 331 patients with advanced MTC that had spread to other parts of the body, and showed a 54 percent reduction in risk for disease progression with Caprelsa compared to placebo.

“Advanced Medullary Thyroid Cancer is a rare disease with a poor prognosis and clinical outcomes for patients with advanced MTC have not changed substantially in the past 20 years. CAPRELSA offers a new treatment option for these patients. It is the first advance of its kind for this devastating disease and we are delighted CAPRELSA has been approved for use in Europe,” said James Vasselli, CAPRELSA Medical Science Director.

Caprelsa, also known as Vandetanib, works through blocking vascular endothelial growth factor receptor and the epidermal growth factor receptor. The drug also targets RET-tyrosine kinase, an important growth driver in certain types of thyroid cancer.

Vandetanib was approved by the U.S. FDA in April 2011 for treatment of late-stage (metastatic) medullary thyroid cancer in adult patients who are ineligible for surgery. It is also approved in Canada and is under review in Russia, Switzerland, Brazil, Mexico, Argentina and Australia

About 45,000 cases of thyroid cancer will be diagnosed this year in the U.S., according to the American Cancer Society. Across Europe, there are around 48,000 cases per year. Around 25% of all MTC cases are genetic in nature, caused by a mutation in the RET proto-oncogene.

Vandetanib was once regarded as a potential blockbuster for the London-based AstraZeneca, but suffered a major setback after failing to extend survival in patients with lung cancer.

Roche’s Skin Cancer Drug Zelboraf (vemurafenib) won European Commission approval as a monotherapy for melanoma patients whose tumours express BRAF V600 gene mutations.

The Swiss pharma group’s novel treatment for melanoma, the most aggressive form of skin cancer, has already approved in the U.S. last August. Zelboraf, as a personalised cancer therapy targeted only those patients who test positive for BRAF V600 mutations using the Roche cobas 4800 BRAF V600 Mutation Test.

The EC green light for Zelboraf was well expected as the regulator’s Committee for Medicinal Products for Human Use has endorsed the drug to be granted marketing authorisation in Europe last December.  The drug won FDA approval for the treatment of late-stage melanoma on August 17, 2011.

The European approval is supported data from clinical study that showed Zelboraf improved progression-free survival by about 4 months when compared to chemotherapy.  It also increased overall survival by about 3 months in patients who tested positive for BRAF V600 mutations.

Dr Hal Barron, chief medical officer and head of global product development at Roche, said: “Zelboraf significantly improves patient survival and exemplifies the benefits that Roche’s personalised approach to medicine can provide for patients, physicians and society.”

Zelboraf, also known as Vemurafenib, interrupts the  B-Raf/MEK/ERK pathway when the B-Raf protein has the common V600E mutation, which means the residue number 600 valine on the B-Raf protein is replaced by glutamic acid. Study shows about 60% of melanomas have this mutation.

Zelboraf was co-developed by Plexxikon, now part of the Daiichi Sankyo Group, and Roche under a collaboration agreement.

The U.S. FDA has granted Corcept Therapeutics the right to market Korlym as treatment of Cushing’s Syndrome, a rare hormone disorder caused by excess amount cortisol in the blood.

The Menlo Park, CA-based Corcept said the U.S. regulators cleared Korlym, also known as Mifepristone which has the same ingredient as the abortion pill RU-486, to treat patients with endogenous Cushing’s syndrome along with type 2 diabetes or glucose intolerance, and are not applicable to surgeries. Korlym is the first drug approved by FDA for the indication, according to the U.S. agency.

Cushing’s syndrome, characterized by central obesity, high blood sugar, insulin resistance,  hypertension and skin problems, is a serious, debilitating condition that usually affects adults between the ages of 25 and 40. Endogenous Cushing’s syndrome is caused by the overproduction of cortisol by the adrenal glands. It is estimated that Cushing’s syndrome affects about 20,000 patients in the U.S. and FDA says nearly 5,000 patients will be eligible for treatment with newly-approved Korlym, which was granted orphan drug designation by the agency.

Robert L. Roe, Corcept’s President, said: “Korlym is a significant advance in the treatment of patients suffering from the debilitating symptoms of Cushing’s syndrome. For the first time, these patients have access to an approved therapy when surgery has failed or is not an option.”

FDA’s approval of Korlym was based on the results of a phase III study of 50 patients who had endogenous Cushing’s syndrome and were either not eligible for or had relapsed from surgery.  It showed that 60 percent of patients with glucose intolerant had a greater than 25 percent improvement in the test after the Korlym treatment.  A separate open-label extension of the clinical trial is still ongoing.

Korlym, as a glucocorticoid receptor and progestin receptor antognist, does not reduce the cortisol production directly; it works through blocking the binding of cortisol to its receptor, therefore  prevent the effects of cortisol. The drug has the common side effects such as nausea, fatigue, headache, arthralgia, vomiting, swelling of the extremities, dizziness and decreased appetite.

As the active ingredient of Korlym is mifepristone, which is controversially indicated for the termination of early pregnancy, the drug will carry a boxed warning that it will terminate a pregnancy.

Corcept said the once-daily pill Korlym will be available  through a small number of endocrinologists who treat patients affected with Cushing’s syndrome from the first of May. The company also said it will control the distribution of the drug to reduce the potential use for ending a pregnancy.

In Europe, Novartis is developing Pasireotide, an somatostatin analog, for the same indication. The experimental drug, known by its brand name Signifor gained European Committee’s support to treat patients suffering from Cushing’s syndrome last month.

The U.S. FDA has given green light to Merck for its eye drug Zioptan to reduce pressure within the eye in people with high blood pressure of the eye (ocular hypertension) or open-angle glaucoma, the most common form of glaucoma.

Merck’s Zioptan, a 0.0015 percent solution of tafluprost ophthalmic, is a prostaglandin analog for lowering high intraocular pressure (IOP).  The company says Zioptan is the first preservative-free prostaglandin analogue ophthalmic solution approved for this indication. 

“Prostaglandin analogs are often used as a first line of treatment to lower intraocular pressure in patients with open-angle glaucoma. The approval of ZIOPTAN will provide a new, effective option to lower IOP,” said George L. Spaeth, M.D., Wills Eye Institute, Philadelphia, “I anticipate using ZIOPTAN in many of these patients in my practice.”

FDA’s approval was based on the safety and efficacy results from five clinical studies involved 905 patients.  The results demonstrated that once-daily Zioptan lowered IOP at three and six months by 6-8 mmHg and 5-8 mmHg, respectively, from a baseline pressure of 23-26 mmHg. 

The most common side effects included increased pigmentation of the iris and eyelids, and changes in the thickness and color of the eyelashes. Merck says the eysdrops should not be used by children or by pregnant women.

People with high intraocular pressure are at a higher risk of developing glaucoma, which is when the optic nerve is damaged and may lead to blindness if left untreated. Open-angle glaucoma accounts for 90% of glaucoma cases in the United States.

 The tafluprost ophthalmic solution has already been approved in some European countries for the indication under the brand Saflutan. Merck said the drug was originally rejected by US regulators late last year, but should now be available throughout the US in March. The drug will be priced at $97 for a 30-day supply.

The U.S. FDA has approved Eli Lilly and Boehringer Ingelheim’s Jentadueto, a new combination medicine for type 2 diabetes.  

Jentadueto is a a single-tablet combination pill containing the DPP-4 inhibitor, linagliptin and metformin. The twice daily pill can be used alone or in combination with a sulfonylurea. The drug has not been studied in combination with insulin and therefore it is not known if it is safe and effective when used with insulin. Jentadueto is not for the treatment of patients with type 1 diabetes or with increased ketones in the blood or urine.

The DPP-4 inhibito linagliptin, one of the two active ingredients in Jentadueto, was approved by FDA approval in May 2011 for improving blood glucose control in adults with type 2 diabetes, either as a stand-alone or in combination with other therapies.

The approval was based on the data from a 24-week, randomized, double-blind, placebo-controlled study that  involved 791 adults with type 2 diabetes inadequately managed with diet and exercise.  It is demonstrated that Jentadueto could lead to placebo-corrected reductions in hemoglobin A1c levels of up to 1.7 percentage points.

 Eli Lilly and Boehringer said the most common side effects with the combo drug are nasopharyngitis and diarrhea. They also note that hypoglycemia was more commonly reported in patients treated with the drug combination and sulfonylurea compared with those treated with the combination of placebo, sulfonylurea, and metformin.

The U.S. FDA has approved Vertex Pharmaceuticals’  Kalydeco as the first drug to treat the underlying cause of cystic fibrosis rather than the symptoms.

Vertex Pharmaceuticals was granted the rights by the U.S. agency to market Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. The approval came three months ahead of the expected deadline.

Cystic fibrosis was caused by a mutation in the CFTR gene, which is required to regulate the components of sweat, digestive juices, and mucus.  This most-common fatal genetic disease in the Caucasian population affects about 30,000 patients in the U.S. and nearly 70,000 people worldwide. There are about 4 percent of them have the G551D mutation.  Cystic fibrosis can produce a thick mucus in the lungs and digestive tract, leading to infections, digestive problems and death in young adulthood. 

Previously cystic fibrosis patients could only be treated with medicines that only tackled the symptoms of the disease, such as antibiotics to treat the infections. Kalydeco, however, is designed to a correct a specific genetic defect and works on a defective protein that acts as a chloride-pump in cell membranes to function properly, restoring the proper flow of water in the body.

Kalydeco is part of a growing number of new medicines that target rare genetic variations found in subgroups of patients.  Dr. Margaret A. Hamburg, the FDA Commissioner, said in a statement: “Kalydeco is an excellent example of the promise of personalized medicine — targeted drugs that treat patients with a specific genetic makeup.” 

In clinical trials, Kalydeco significantly improved lung function, reduced the number of lung problems that required treatment with antibiotics and led to weight gain, which is desirable for CF patients who typically have trouble maintaining a healthy weight. The U.S. agency reviewed Kalydeco under fast-tracked process that was granted to medicines that represent a significant advance over existing therapies.

Kalydeco was approved for use in patients six-years-old and older. Vertex is planning a study of the drug in patients between two years and five years old. Kalydeco works on patients with G551D gene mutation. Vertex is developing Kalydeco in combination with another drug candidate to address that problem with majority of cystic fibrosis patients that have a different genetic defect.

The most-common side effects of Kalydeco include upper respiratory tract infection, headache, stomach ache, rash, diarrhea and dizziness, according to the FDA.

Vertex said treatment with Kalydeco costs $294,000 per year. The price tag put Kalydeco on par with the most expensive therapies on the market. Vertex said the company will also have a variety of patient assistance programs to help those unable to afford the potentially life-altering treatment.

Kalydeco is also under a priority review by European regulators.Vertex said if all goes well European approval could come in the third quarter of 2012.

Pfizer’s Inlyta (axitinib) gains the U.S. FDA approval as a second-line treatment for patients with advanced renal cell carcinoma (RCC), the most common type of  kidney cancer.

The U.S. regulators made the decision, following the recommendation of an FDA advisory panel, to give green light to Pfizer’s drug candidate axitinib to treat patients with advanced kidney cancer who have not responded to first-line treatments.  According to Pfizer, Inlyta is the first treatment to demonstrate superior benefit in a phase III study compared with another targeted agent in advanced RCC.

The FDA’s approval of Inlyta is based on data from the randomize, multi-center, open-label Phase III trial that involved 723 patients, who experienced cancer progression during or after treatment with at least one systemic therapy.  Data from the late stage study indicated that Inlyta extended progression-free survival (PFS) by 6.7 months in patients with advanced RCC, while  Bayer/Onyx’s Nexavar (sorafenib), the standard treatment, extended PFS by 4.7 months.

Renal cell carcinoma is the most common type of kidney cancer in adults, responsible for approximately 80% of cases. RCC is the most common form of kidney cancer, with 51190 new cases diagnosed in the U.S. and 12890 deaths reported in 2007.  Despite several new targeted therapies entered the market in recent years,  five-year survival rates of RCC remain low when metastases have spread.

Inlyta is the seventh drug approved for dvanced renal cell carcinoma since 2005, noted Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

“Collectively, this unprecedented level of drug development within this time period has significantly altered the treatment paradigm of metastatic kidney cancer, and offers patients multiple treatment options,” said Richard Pazdur. The FDA approved treatment of kidney cancer include sorafenib from Bayer and Onyx (2005), sunitinib (Sutent) from Pfizer (2006), temsirolimus (Torisel) from Pfizer-Wyeth (2007), everolimus from Novartis (2009),   bevacizumab (Avastin) from Roche (2009) and  pazopanib (Votrient) from GSK (2009).  

“Pfizer has a strong commitment to advancing therapies for patients with advanced RCC. INLYTA is an important addition to our portfolio of treatment options for these patients, which also includes Sutent (sunitinib) and Torisel (temsirolimus),” said Garry Nicholson, president and general manager, Pfizer Oncology Business Unit.

The U.S. agency said that hypertensive patients should have their blood pressure well-controlled before taking Inlyta. Some patients had bleeding problems, and some of those died as a result of the bleeding. Other side effects of Inlyta included vomiting, weight loss, nausea, reduced appetite, constipation, weakness, dysphonia (voice loss),  fatigue and hypertension.

The FDA also started on the same day to review Pfizer’s NDA for bosutinib as a treatment for patients with Philadelphia chromosome-positive chronic myeloid leukaemia after they failed second-line therapy with Bristol-Myers Squibb’s Sprycel (dasatinib) or Novartis’ Tasigna (nilotinib).

Roche’s skin cancer drug Erivedge was approved by the U.S. FDA to treat patients with a common form of skin cancer, basal cell carcinoma (BCC).

Erivedge, also named as vismodegib,  is the first drug available to treat basal cell cancer that has spread locally or metastasized to distant parts of the body. The drug was developed by Roche’s biotech unit, Genentech and its partner Curis Inc. of Lexington, MA.

The U.S. regulators granted green light to the once-a-day pill, Erivedge, based on the results from ERIVANCE BCC Phase II trial that enrolled 104 patients with advanced BCC. The study achived its primary endpoint by showing Erivedge shrank lesions in 27 of 63 patients with locally advanced BCC and 10 out of 33 patients with metastatic BCC.

The approval comes ahead of schedule under a fast six-month review process as the FDA said that currently there were no approved treatments for basal cell carcinoma.

Hal Barron, chief medical officer and head, Global Product Development at Genentech, said:“Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions.” 

“We are pleased that in the last six months we have been able to provide two new medicines for different types of advanced skin cancer to people who previously had few or no treatment options,” Hal Barron added.

Basal cell carcinoma is considered the least dangerous of the three types of skin cancer: basal cell carcinoma, squamous cell carcinoma and melanoma. It is usually restricted to a small area of the skin, but in the rare cases it can progress to advanced stage that often lead to metastasize and causing severe deformity or loss of function of affected organs.  BCC is the most common skin cancer, affecting more than 2 million people in the U.S. in 2010.

Erivedge is the first approved drug targeting Hedgehog pathway, which is pathogenetically relevant in more than 90% of basal-cell carcinomas. The drug works by blocking the smoothened receptor of this signaling pathway, and prevents the expression of tumor mediating genes within the pathway.

 “Our understanding of molecular pathways involved in cancer, such as the Hedgehog pathway, has enabled the development of targeted drugs for specific diseases,” said Richard Pazdur, director of the office of haematology and oncology at the FDA, “This approach is becoming more common and will potentially allow cancer drugs to be developed more quickly. This is important for patients who will have access to more effective therapies with potentially fewer side effects.”

Erivedge was approved with a Black Box safety warning that the drug is linked to fetal death and severe birth defects when it is used by pregnant women.  Other side effects of Erivedge include muscle spasms, hair loss, change in how things taste or loss of taste, weight loss, tiredness, nausea, diarrhea, decreased appetite, constipation, vomiting and joint aches.

As the first drug to be approved for BCC  in the US, Erivedge will cost $7,500 a month and the total cost is expected to be around $75,000 for a 10 month treatment course.

Erivedge would be available in the US  within one to two weeks, according to Genentech. The company said it has also submitted a marketing authorisation for Erivedge in Europe.

The Japanese pharma company Takeda got the green light to market its high-blood pressure treatment Edarbi in Europe, according to the company’s statement.

The European Commission has approved Edarbi, also known by the generic name Azilsartan Medoxomil, for the treatment of essential hypertension (high blood pressure) in adults. As one of the class “sartan” compound, the drug is a once-daily angiotensin receptor blocker, also known as angiotensin II receptor antagonist. Angiotensin is a peptide hormone that can cause blood vessels to contract, and lead to increased blood pressure.

Takeda say it will launch Edarbi across Europe in 2012 starting with Germany in January.

Trevor Smith, Takeda’s head of Europe and Canada, said,”The marketing authorization for azilsartan medoxomil marks an important milestone for Takeda, building on our 30-year heritage in cardiovascular disease and reinforcing our commitment to expand the boundaries of hypertension treatment, address unmet needs and ultimately optimize patient outcomes across Europe”.

European Medicines Agency’s advisory panel gave a positive opinion for Edarbi based on the results from an extensive pre-clinical and clinical development program, including seven phase III clinical trials involving nearly 6,000 patients with essential hypertension. The late-stage studies showed Edarbi’s superiority for lowering blood pressure at 80 mg/day to placebo and other two commonly-prescribed angiotensin receptor blockers, olmesartan at 40 mg per day, and valsartan at 320 mg per day.

FDA has approved azilsartan medoxomil for the treatment of high blood pressure in adults earlier this year.