Approvals hit a new all-year high of 8 innovative drugs in August

In August 2020, the U.S. Food and Drug Administration (FDA) opened a full-time high for the approval of innovative drugs, approving a total of eight innovative drugs, including five new molecular entities (NDEs) and three new biological products.
January-August 2020, the FDA has approved 38 innovative drugs, and the number of innovative drugs approved throughout the year is expected to exceed 2019.
Figure 1. Number of new drugs approved by the FDA in the last decade Source: FDA website, public information August FDA approved eight innovative drugs covering a number of disease areas, including a number of breakthrough drugs, such as the world's first approved anti-BCMA (B-cell mature antigen) antibody association drug Blinrep (belantamab mafodotin); The oral small molecule drug Evrysdi (risdiplam); the first external androgen-receiving inhibitor for acne, Winlevi(clascoterone); the first antibody drug, Ensralizumab, which targets adult patients with AQP4-positive neurospinal cord disease.
the biggest winner in August, with two heavy-duty drugs approved.
market with Spinraza and Zolgensma after the launch of Evrysdi, the world's third-largest drug for spinal muscular dystrophy, the industry has high expectations for Evrysdi, with EvaluatePharma predicting sales of $803 million by 2024.
another single anti-drug, satralizumab, which treats neuroscolonitis spectrum disorders, has been developed in many countries and regions around the world and has been approved for listing in Canada, Japan, Switzerland and the United States.
Table 1. FDA Approval of New Drugs Source: THE FDA website, belantamab mafodotin-blmf August 5, GlaxoSmithKline announces that the FDA has accelerated approval of its development of Blenrep (belantamab) mafodotin-blmf) is available as a monotherapy for the treatment of adult patients with relapsed or refractic multiple myeloma (MM) who have previously received at least 4 therapies (including anti-CD38 antibodies, protease inhibitors, immunomodulants).
previously, Blenrep had been eligible for FDA-granted breakthrough therapies, and its new drug applications had been prioritized.
Blenrep is the world's first approved anti-BCMA (B-cell mature antigen) antibody association drug (ADC) with the potential to improve treatments for patients with relapsed or refrogenic myeloma today.
, Blenrep has also received conditional EU approval to treat patients with multiple myeloma.
Figure 2. Belantamab mafodotin Specific source of information: Minet Global Drug Development Library Multiple myeloma is the most common type of malignant plasma cell disease, characterized by unbridled growth and extensive immersion of malignant plasma cells in the bone marrow.
, an estimated 32,000 people have been diagnosed with multiple myeloma this year, and nearly 13,000 will die from the disease.
incidence of multiple myeloma in China is slightly lower than in Europe and the United States, but the trend of increasing year by year is obvious.
the safety and ver effectiveness of Blenrep has been proven in a Phase II clinical trial called Dramm.
196 eligible patients with multiple myeloma were randomly treated in two groups, and the results showed that the total remission rate (ORR) of 2.5 mg/kg single-drug therapy was 31% every three weeks, the medium duration of remission (DOR) had not yet been achieved, but 73% of patients (respondents) had DOR≥6 months.
since the FDA approved three ADC drugs in a row in 2019, the ADC has fought a "turning the page" and is now a hot global research field.
year, 10 ADC drugs have been approved for sale worldwide, including belantamab mafodotin, and trastuzumab emtansine is the first ADC drug to go on sale in China in January.
Many domestic pharmaceutical companies have also laid out ADC drugs, August 27, Rongchang biological injection with the wei dixito monoanti (commodity name: Edish) new drug market application was formally accepted, and was included in the priority review process, for the treatment of local late stage or metastatic gastric cancer (including gastroesophageal adenocarcinoma) patients.
is the first self-developed ADC drug in China to submit a new drug for market.
Table 2. On August 6th, the FDA accelerated approval of Bayer's Nifurtimox for the treatment of pediatric patients with Chagas disease caused by a trypanosomiasis infection.
Lampit is a new formula, secondiable, easily dispersed tablets that can be dispersed in water and then given, greatly convenient for young or difficult to swallow newborns, infants and children.
3. Nifurtimox Source: Minenet Global Drug Research and Development Kuchagas disease, also known as Trypanosomiasis, is endemic mainly in the Latin American continent and has affected the health of 6 to 7 million people.
is often overlooked because its clinical processes develop slowly and often asymptomaticly, so that patients are often diagnosed at a later stage, so they are known as "silent diseases".
if left untreated, Chagas disease can lead to severe heart and digestive tract lesions and can be fatal.
FDA's approval of Lampit for pediatric patients is a major advance in controlling the spread of the disease.
's outstanding performance in Phase III clinical trials provides the basis for this approval.
CHICO study of 330 child patients, two-thirds of the child patients received a 60-day Lampit treatment programme, while the remaining patients received a 30-day Lampit treatment programme and followed up for one year after treatment.
results showed that the 60-day Lampit program was superior to the historical placebo control and reached the main endpoint of the study in terms of serological response followed up for one year after treatment.
across the study group, the 60-day programme has advantages over the 30-day programme (unapproved administration programme) in serological response.
7 (Risdiplam), the FDA approved the listing of Evrysdi, a Roche subsidiary, for the treatment of children and adults with spinal muscular dystrophy (SMA) of 2 months and older.
Evrysdi is a liquid preparation that can be delivered at home by oral or nasal feeding.
the world's first oral small molecule drug to treat spinal muscular dystrophy, and the only home-given drug to treat spinal muscular dystrophy.
, Evrysdi has been granted FDA-granted orphan drug eligibility, and its new drug applications have been fast-tracked and prioritized.
, Evrysdi has submitted listing applications in several countries, including Brazil, South Korea and China.
Figure 4.risdiplam Specific sources of information: Minenet Global Drug Development Library Spinal myotrophic dystrophy is a rare neurodegenerative disease with recessive inheritance of the common chromosome, with a incidence rate of approximately 1/6000-1/10000 in newborns.
it can lead to the gradual deterioration of the nerves that control the muscles, muscle atrophy, and eventually most patients die of respiratory muscle failure.
two studies, FIREFISH and SUNFISH, laid a solid foundation for Evrysdi's approval.
FIREFISH and SUNFISH studies, 41 percent of children were treated with Evrysdi to be able to sit alone, most infants were able to survive longer without permanent breathing, and post-treatment children and adults experienced clinical and statistically significant improvements in motor function.
August, the news that "a rare disease drug sells 700,000" was swiped by the public and posted on Weibo, saying that the "rare disease" was SMA, and that "drug" was the world's first drug to treat SMA drugs, Spinraza.
Spinraza (Nosinas sodium injection) was approved by the State Drug Administration in 2019, is currently the only treatment of SMA drugs, because has not yet entered the health insurance, the current price of about 700,000 yuan a shot, the first year of purchase costs about 4.2 million, after 2.1 million a year.
only three SMA drugs are currently on the market worldwide, and the approved Bayer Evrysdi is in the process of submitting an application for listing at home, which, if approved, will break the single situation in the domestic SMA market and give SMA patients more options.
Table 3. SMA Drug Information Oliceridine August 7, the FDA announced the approval of Trevena's painkiller Olinvyk (oliceridine) for the treatment of patients with pain so severe that intravenous opioids, alternative therapies do not adequately control pain in moderate to severe acute pain adult patients.
Olinvyk is an opioid agitant, and its active ingredient, olicridine, is the first intravenous analgesic.
, Jiangsu Enhua Pharmaceuticals, as Trevena's only partner in China, has an exclusive license agreement to develop and commercialize Olinvyk in China.
, Enhua Pharmaceuticals has been approved by the State Drug Administration to begin clinical trials of Olicridine fumarate injections.
Figure 5. Oliceridine Source: Minernet Global Drug Research and Development Library opioids have superior pain-relief effects, but at the same time the abuse and addiction of opioids has become a public health crisis in the United States.
In this environment, Olinvyk can still be approved, on the one hand, relying on its unique and differentiated characteristics in pharmacodynamics: (1) rapid and long-lasting efficacy, most patients feel pain relief 2-5 minutes after administration, the efficacy lasts about 3 hours.
(2) there is no known active metabolism, and (3) in elderly patients or patients with impaired renal function, dose adjustment is not required, providing a new option for at-risk patients.
, on the other hand, is its excellence in clinical trials: a total of 1,535 patients who underwent Bunion surgery or abdominal surgery participated in clinical trials, and data showed that patients who took oral Olinvyk were effectively reduced in pain at approved doses compared to the placebo group.
Olinvyk is similar to other opioids.
Viltolarsen, the U.S. FDA accelerated approval of Viltolarsen, a subsidiary of NS Pharma, a subsidiary of Japan's New Drug Co., Ltd., on August 12 for the treatment of patients with Du's muscular dystrophy (DMD) who have been diagnosed with 53 gene jump mutations in exons.
this is the second FDA-approved targeted treatment for such mutations and the only exon 53 jump treatment that has been shown to increase antimyostrophy protein levels in children as young as 4 years of age.
Viltepso was approved in Japan in March 2020.
Figure 6. Viltolarsen Specific Source: Milnet Global Drug Development Kudu's muscular dystrophy is a common x-chain recessive genetic muscle disease caused by a mutation in the DMD gene that causes the loss of antimyostrogen, leading to degenerative muscle degeneration, necrosis and atrophy.
incidence of Duchy muscular dystrophy in the general male population, in our country, the incidence of Duchy muscular dystrophy is about 1/3500.
Viltepso's approval is based on two clinical trials in North America and Japan, respectively.
results showed that 100% of patients showed an increase in antimyostrophy protein levels after treatment with Viltepso, and 88% showed an increase in antimyostrogen levels of 3% or higher than normal.
20-24 weeks of treatment with Viltepso (80mg/kg/wk), the patient's antimyostrophy protein level increased to 6% of its normal value, and the level of antimyostrogen was 10 times higher than before treatment.
August 14th the FDA approved the listing of Streizumab-mwge, a roche subsidiary of Gene Tank, for the treatment of adult patients with anti-water channel protein-4 (AQP4) antibody-positive optic neurone spinal cord spectrum disorder (NMOSD).
Enspryng is the first and only antibody drug approved for the treatment of AQP4 antibody-positive NMOSD adult patients with targeted suppression of lecytocyte mesotonin-6 subjects (IL-6R).
comes after Enspryng was awarded a breakthrough drug by the FDA.
Figure 7. satralizumab Specific sources of information: Mienet Global Drug Research and Development Library NMOSD is a rare, severe autoimmune disease of neuritis, which is mediated by human B cells and attacks the optic nerve, spinal cord and brain trunk, in addition to potentially irreversible insomnia and paralysis, patients may also experience sensory loss, bladder and bowel dysfunction, nerve pain and respiratory failure.
the approval is based on two clinical trials called SakuraStar and SAkuraSky.
SakuraStar study evaluated the efficacy and safety of Enspryng monotherapy compared to placebos, with results