Alexion Pharmaceuticals Inc

Kanuma was acquired by Alexion Pharmaceuticals on completion of the acquisition of Synageva Pharmaceuticals in June 2015.

WTX101 has a unique action mechanism. The drug has the potential to become the standard of care for Wilson disease patients.

Horizon plans to drive Krystexxa through increased awareness and outreach, investing in its marketing and medical education as well as commercial infrastructure.

In November 2018, of the total 18 analysts covering Alexion Pharmaceuticals (ALXN), 16 analysts have given the stock a “buy” or higher rating, and two analysts have given it a “hold” rating.

In 4Q17, Pfizer’s BeneFix reported revenues of $150 million, which represents a ~11% decline YoY and a ~1% decline quarter-over-quarter.

In 4Q17, Biogen’s (BIIB) Spinraza generated revenues of $363 million, which reflected 34% quarter-over-quarter growth.

Analysts expect Incyte Corporation’s revenue to rise ~26.7% to $413.8 million in 4Q17 compared to $326.5 million in 4Q16.

On September 7, 2017, Alnylam Pharmaceuticals (ALNY) announced that it had reached an agreement with the US Food and Drug Administration (or FDA) related to the design of the phase three program for investigational RNAi therapeutic Givosiran.

In 2Q17, Jazz Pharmaceuticals’ (JAZZ) Defitelio generated revenues of ~$30 million, which represents a ~10% fall YoY and a ~17% fall QoQ.

In June 2017, the FDA accepted Bioverativ’s investigative new drug application for BIVV001, a drug designed to treat prophylaxis from bleeding associated with hemophilia A.

Since demand from the target population for prophylaxis is rising rapidly, Bioverativ’s Alprolix and Eloctate are expected to see solid demand trends in 2017.

Bioverativ (BIVV) expects its 2017 GAAP and non-GAAP operating margins to fall 38.0%–42.0% and 43.0%–47.0%, respectively.

In 1Q17, Bioverativ reported revenues close to $259.0 million, driven by its focus on a commercial strategy for its hemophilia products and optimal cost management.

The acquisition of True North Therapeutics has paved the way for Bioverativ’s (BIVV) entry into cold agglutinin disease (or CAD).

Vertex Pharmaceuticals’ (VRTX) drugs, Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor), are capable of treating around 30,000 cystic fibrosis (or CF) patients.

Alexion Pharmaceuticals has planned five clinical trials in 2017 to test the potential of investigational next-generation C5 antibody, ALXN1210, as a treatment option for complement-mediated diseases.

To promote the use of Spinraza for SMA, Biogen (BIIB) has been actively educating and creating awareness for the drug among physician and patient communities.

Vertex Pharmaceuticals (VRTX) is aiming to increase the number of patients eligible to be treated with its drugs to include the entire CF patient population.

In January 2012, Gilead Sciences (GILD) acquired Pharmasset and got access to sofosbuvir. In December 2013, the FDA approved sofosbuvir under the brand name Sovaldi.

Alexion (ALXN) has been serving the atypical hemolytic uremic syndrome, or aHUS, market for the past five years.

Vyvanse is Shire’s leading ADHD drug, constituting 80% of the company’s ADHD portfolio. In fiscal 2015, Vyvanse earned the company $1.7 billion.

Gattex is the first and only analog of glucagon-like peptide-2 (or GLP-2) indicated for short bowel syndrome. The drug is known as Gattex in the United States and Revestive in Europe.

Kuvan, with the active ingredient sapropterin dihydrochloride, is effective in reducing blood phenylalanine levels in PKU (or phenylketonuria) patients.

The wholesale cost per patient for Naglazyme is around $485,747 per year. The drug has been effective in improving walking and stair-climbing capacity.

Alexion Pharmaceuticals’ Kanuma is an innovative enzyme replacement therapy for patients suffering with lysosomal acid lipase deficiency (or LAL-D).

Mature biotechnology companies generally earn EBITDA margins of about 30% to 40%.

While mature biotechnology companies with drugs in multiple disease segments earn an average of 30%–40% EBITDA, margins of companies targeting only rare diseases can vary due to unique business models.

Alexion Pharmaceuticals (ALXN) has strengthened its drug pipeline by diversifying its research programs across the metabolic disorder segment.

As part of its research and development, Vertex Pharmaceuticals (VRTX) has been actively exploring new cystic fibrosis (or CF) drugs as well as other indications for its existing drug Kalydeco.

Alexion Pharmaceuticals is actively involved in expanding the approved labels for its flagship product Soliris.

On July 2, 2015, the FDA (U.S. Food and Drug Administration) approved Orkambi, a combination drug of lumacaftor and ivacaftor, for treating cystic fibrosis (or CF) patients.