生物医药系列产品
数据
资源
版本对比
免费注册
预约演示
免费注册
FDA Action Alert: Blueprint, Opiant and Lexicon
2023-05-22
·
BioSpace
临床3期
申请上市
上市批准
临床结果
优先审批
FDA Action Alert logo/© Nicole Bean for BioSpace The FDA is set to decide on three drug applications this week, including ones for opioid overdose,
heart failure
and
systemic mastocytosis
. Read below for more. Blueprint Seeks Approval in Indolent
Systemic Mastocytosis
On May 22, the FDA is expected to reach a regulatory decision on Blueprint Medicines’
Ayvakit
(
avapritinib
), an investigational kinase inhibitor being proposed for the treatment of
indolent systemic mastocytosis (SM)
. SM is a rare, genetic condition characterized by the frenzied and uncontrollable activation and proliferation of mast cells, a type of white blood cell. Most SM patients have non-advanced—or indolent—disease, which is associated with a broad range of severe, chronic and often unpredictable symptoms such as
anaphylaxis
,
pruritus
,
diarrhea
,
fatigue
and
bone pain
. Patients with SM often suffer from poor quality of life due to the fear of developing unexpected symptoms. There are currently no approved treatments for
indolent SM
. In approximately 95% of cases, SM is caused by the D816V mutation in the
KIT
gene.
Ayvakit
works by targeting and binding to the mutant protein, thereby disrupting its downstream signaling cascade. In this way,
Ayvakit
suppresses the proliferation of mast cells bearing the D816V mutation. Blueprint backed Ayvakit’s supplemental NDA with data from the PIONEER trial, which found that the drug significantly and rapidly reduced all main measures of pathological mast cell compared to placebo. These effects were durable throughout 24 weeks of follow-up. Moreover,
Ayvakit
treatment led to significant and clinically meaningful improvements in quality of life, Blueprint reported. PIONEER is the largest randomized and placebo-controlled study in indolent SM.
Ayvakit
is already approved for
advanced SM
, SM with associated
hematological neoplasms
,
aggressive SM
,
mast cell leukemia
and
unresectable or metastatic gastrointestinal stromal tumor
.
Opiant
’s Overdose Spray Awaits Verdict Also on May 22, the FDA is expected to make a decision on
Opiant Pharmaceuticals
’ nasal spray candidate for opioid overdose,
OPNT003
.
OPNT003
is
Opiant
Opiant
’s nasal spray formulation for
nalmefene
, an
opioid antagonist
that blocks
opioid receptors
to achieve partial or complete reversal of overdose effects, such as
hypotension
and
respiratory depression
. Data from three clinical studies were used to support
Opiant
Opiant
’s NDA. The first study compared
OPNT003
’s pharmacokinetic pro an intramuscular
nalmefene
injection and found that the nasal route of administration leads to faster absorption and higher plasma concentrations of the drug.
Opiant
ran another pharmacokinetic study looking at the different effects of administering
OPNT003
in one or both nostrils, showing that even when administered as multiple doses, the candidate is safe and well-tolerated. The company also submitted data from a head-to-head pharmacodynamic study comparing
OPNT003
against Emergent BioSolutions’
Narcan (naloxone HCl)
nasal spray. This study found that
Opiant
Opiant
’s nasal spray was not only non-inferior to
Narcan
but also induced nearly twice the reversal in
opioid-induced respiratory depression
. The California-based biotech completed its rolling submission for the NDA in November 2022, which the FDA accepted in January 2023, granting it Priority Review designation.
Lexicon
Proposes
Sotagliflozin
for
Heart Failure
Treatment The FDA’s final decision of the week, which it is scheduled to release by May 27, pertains to
Lexicon Pharmaceuticals
’
sotagliflozin
for the treatment of
heart failure
.
Sotagliflozin
is an orally available drug that works by inhibiting both the
SGLT1
and SGLT2 proteins, which play a role in glucose absorption in the gastrointestinal tract and kidneys, respectively. In its NDA, accepted by the FDA in July 2022,
Lexicon
is seeking approval of
sotagliflozin
to reduce the risk of cardiovascular death, hospitalization for
heart failure
and urgent
heart failure
visits in adult patients. The company is also proposing to use
sotagliflozin
to decrease the risk of these outcomes, along with
nonfatal myocardial infarction
and
stroke
, in adults with
chronic kidney disease
,
type 2 diabetes
and other cardiovascular risk factors.
Lexicon
supported its NDA with data from the randomized, double-blinded and placebo-controlled Phase III SOLOIST-WHF and SCORED studies. SOLOIST-WHF looked at the efficacy and safety of
sotagliflozin
in more than 1,200
type 2 diabetes
patients who had recently been hospitalized for worsening
heart failure
. The study found that the drug significantly reduced
heart failure
events and readmissions. Meanwhile, SCORED enrolled more than 10,000 patients with
type 2 diabetes
,
chronic kidney disease
and risk factors for
cardiovascular disease
. Its results showed the drug significantly lowered rates of
heart failure
hospitalizations, cardiovascular deaths and urgent visits. Aside from
heart failure
,
sotagliflozin
is being assessed for
chronic kidney disease
and
type 1 and type 2 diabetes
. The drug is approved in the European Union for
type 1 diabetes
under the brand name
Zynquista
. Tristan Manalac is an independent science writer based in metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com
更多内容,
请访问原始网站
文中所述内容并不反映新药情报库及其所属公司任何意见及观点,如有版权侵扰或错误之处,请及时联系我们,我们会在24小时内配合处理。
机构
Opiant Pharmaceuticals, Inc.
Lexicon Corp.
Lexicon Pharmaceuticals, Inc.
适应症
心脏衰竭
系统性肥大细胞增多症
髓性系统性肥大细胞增多症
[+19]
靶点
c-Kit
Opioid receptors
SGLT1
药物
阿伐替尼
盐酸纳美芬
OPNT-005
[+4]
标准版
¥
16800
元/账号/年
新药情报库 | 省钱又好用!
立即使用
来和芽仔聊天吧
热门报告
特应性皮炎深度解析:药物开发、专利分析与风险评估
智慧芽生物医药
2024年7月全球首批及特殊审评药物报告
智慧芽生物医药
GPRC5D靶点专利调研报告
智慧芽生物医药
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台,助您全方位提升您的研发与决策效率。
开始免费试用
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台,以API或者数据包形式对外开放,助您更加充分利用智慧芽新药情报信息。
试用数据服务