175:(LAL Deficiency). Kanuma is a recombinant form of the natural human LAL enzyme and an enzyme replacement therapy being developed for patients with LAL Deficiency. LAL Deficiency is a serious, life-threatening disease associated with early mortality and significant morbidity. LAL Deficiency is a chronic disease in which genetic mutations result in decreased activity of the LAL enzyme; this leads to marked accumulation of lipids in vital organs, blood vessels, and other tissues, resulting in progressive and multi-systemic organ damage including fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, cardiovascular disease, and other devastating consequences. LAL Deficiency affects patients of all ages with sudden and unpredictable clinical complications manifesting from infancy through adulthood. The disease can be diagnosed with a simple blood test.
140:
devastating, rare diseases. This represented a substantial shift in the original company's business strategy of developing bio-similar drugs using its novel egg white (EW) protein production technology. The company's proprietary technology produces proteins by recombinant DNA technology in egg white (EW) using a transgenic Gallus expression system. The proteins are then purified using conventional recombinant protein chromatographic methods. The EW system can consistently express proteins that contain glycan structures, predominantly GlcNAc- and mannose-terminated N-linked glycan structures, as well as mannose-6-phosphate (M6P) glycans, which are specifically recognized and internalized via receptors into key target cells.
110:
183:
enzyme replacement therapy in preclinical development for disorders of calcification. In addition to these first-mover programs, Synageva's pipeline also consisted of opportunities that leveraged the company's EW manufacturing platform and other capabilities to create potentially bio-superior treatments for patient populations where there is still unmet medical need. The company was able to produce enzymes targeting
159:) and Synageva announced that they entered into a definitive agreement in which Alexion would acquire Synageva for a value of $ 230 per share. The transaction was unanimously approved by both companies’ Boards of Directors and is valued at approximately $ 8.4 billion net of Synageva's cash (total $ 9.1 billion). This was one of the largest premiums paid to any company over $ 5 billion in market cap since 1995.
179:
Deficiency presenting in infants. The FDA accepted for review the
Biologics License Application (BLA) for Kanuma, granted Synageva's request for Priority Review, and established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA). The EMA validated the Marketing Authorization Application (MAA) for Kanuma and granted the company's request for accelerated assessment.
139:
of privately-held
Avigenics, Inc. in Athens, Georgia, a company initially formed by Dr. Robert Ivarie of the University of Georgia. Upon joining AviGenics in June 2008, Mr. Patel created the vision and strategy for Synageva and changed the company's direction to focus on products for the treatment of
126:
dedicated to discovering, developing and delivering medicines for patients with rare diseases and high unmet medical needs. The company had manufacturing and laboratory locations in
Lexington and Holden, Massachusetts, Bogart and Athens Georgia, as well as offices in a variety of locations around the
182:
Synageva's other pipeline programs consisted of protein therapeutic programs for rare diseases with unmet medical need at various stages of development, including a Phase 1/2 trial with its second, first-mover program, SBC-103 for MPS IIIB. The company's third, first-mover program, SBC-105, was an
178:
Kanuma was granted orphan designation by the U.S. Food and Drug
Administration (FDA), the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Kanuma also received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL
143:
With the support from a dedicated and experienced team, Synageva went public on the NASDAQ Global Market in
November 2011 by completing a reverse merger with Trimeris, Inc. Synageva later relocated its corporate headquarters to Lexington, Massachusetts.
220:
Alexion Press
Release. (2015). “Alexion to Acquire Synageva to Strengthen Global Leadership in Developing and Commercializing Transformative Therapies for Patients with Devastating and Rare Diseases”.
265:
260:
250:
135:
Synageva was formed when Sanj K. Patel (formerly an executive at
Genzyme Corporation, Cambridge, Massachusetts) was approached in early 2008 by Baker Bros. Investments to be the
245:
229:
Peter Loftus, Jonathan D. Rockoff and
Maureen Farrell (May 6, 2015). “Alexion-Synageva Deal Shows Lure of Rare-Disease Drugs”. The Wall Street Journal.
255:
270:
211:
Lee
Shearer (April 21, 2014). "UGA BioBusiness Center helps turn discoveries into companies". Athens Banner-Herald. Retrieved May 8, 2015.
172:
123:
82:
148:
136:
109:
53:
184:
168:
28:
239:
192:
188:
156:
36:
195:
with expression levels and activity that supported further preclinical development.
167:
In July 2008, Mr. Patel designed and initiated
Synageva's lead program, Kanuma® (
152:
42:
122:
was a publicly listed biopharmaceutical company headquartered in
98:
171:) to address a rare and devastating disease known as
93:
77:
59:
49:
34:
24:
266:Pharmaceutical companies of the United States
8:
261:Pharmaceutical companies established in 1996
251:Health care companies based in Massachusetts
19:
108:
18:
204:
246:American companies established in 1996
7:
14:
173:lysosomal acid lipase deficiency
256:Companies listed on the Nasdaq
1:
271:2015 mergers and acquisitions
65:; 28 years ago
287:
16:Biopharmaceutical company
124:Lexington, Massachusetts
120:Synageva BioPharma Corp.
83:Lexington, Massachusetts
20:Synageva BioPharma Corp.
149:Alexion Pharmaceuticals
21:
117:
116:
54:Biopharmaceutical
278:
230:
227:
221:
218:
212:
209:
147:On May 6, 2015,
113:
112:
105:
102:
100:
73:
71:
66:
22:
286:
285:
281:
280:
279:
277:
276:
275:
236:
235:
234:
233:
228:
224:
219:
215:
210:
206:
201:
185:Hunter syndrome
169:sebelipase alfa
165:
133:
107:
97:
89:
85:
69:
67:
64:
39:
17:
12:
11:
5:
284:
282:
274:
273:
268:
263:
258:
253:
248:
238:
237:
232:
231:
222:
213:
203:
202:
200:
197:
164:
161:
132:
129:
115:
114:
95:
91:
90:
87:
81:
79:
75:
74:
61:
57:
56:
51:
47:
46:
40:
35:
32:
31:
26:
15:
13:
10:
9:
6:
4:
3:
2:
283:
272:
269:
267:
264:
262:
259:
257:
254:
252:
249:
247:
244:
243:
241:
226:
223:
217:
214:
208:
205:
198:
196:
194:
193:Pompe disease
190:
189:Fabry disease
186:
180:
176:
174:
170:
162:
160:
158:
154:
150:
145:
141:
138:
130:
128:
125:
121:
111:
104:
96:
92:
88:United States
84:
80:
76:
62:
58:
55:
52:
48:
44:
41:
38:
33:
30:
27:
23:
225:
216:
207:
181:
177:
166:
146:
142:
134:
119:
118:
78:Headquarters
25:Company type
45:: GEVA
240:Categories
199:References
101:.synageva
37:Traded as
163:Products
50:Industry
155::
131:History
127:world.
94:Website
68: (
60:Founded
153:Nasdaq
106:
43:Nasdaq
29:Public
191:and
157:ALXN
103:.com
70:1996
63:1996
137:CEO
99:www
242::
187:,
86:,
151:(
72:)
Text is available under the Creative Commons Attribution-ShareAlike License. Additional terms may apply.
