2022H1: Game-changers brought by Eli Lilly, Novartis, etc

Halfway through 2022, the battle for the number of approved new drugs has also ushered in the second half for drug regulators

Halfway through 2022, the battle for the number of approved new drugs has also ushered in the second half for drug regulators

According to statistics, the FDA has approved a total of 16 new drugs in the past 6 months

Although there is no shortage of "epoch-making" new drugs approved in the first half of this year, counting the expanded authorization of some therapeutic indications, it is difficult to deny that the FDA's performance in the number is somewhat mediocre

The optimism is that in the second half of this year, there are also blockbuster products worth looking forward to receiving approval

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2022H1: Game-changers brought by Eli Lilly, Novartis, etc

2022H1: Game-changers brought by Eli Lilly, Novartis, etc.

Cancer is undoubtedly a hot area for the market to bet on, and in 2021, the proportion of new anti-tumor drugs on the market will reach 30%, which more or less responds to the concerns

Cancer is undoubtedly a hot area for the market to bet on, and in 2021, the proportion of new anti-tumor drugs on the market will reach 30%, which more or less responds to the concerns

Among them, there are 4 new anti-tumor drugs, 3 blood diseases and anti-infectious disease drugs, 2 each in the fields of neurological diseases, autoimmune diseases and metabolic diseases, and 1 each for cardiovascular diseases and ophthalmic diseases

Among them, there are 4 new anti-tumor drugs, 3 blood diseases and anti-infectious disease drugs, 2 each in the fields of neurological diseases, autoimmune diseases and metabolic diseases, and 1 each for cardiovascular diseases and ophthalmic diseases

One of the representatives of new anti-tumor drugs is Pluvicto

Globally, prostate cancer is the second most common malignancy in men, after lung cancer
.
Prior to the marketing of Pluvicto, the preferred treatment regimen for patients who were not suitable for radical resection was androgen deprivation therapy, i.
e.
, castration levels
.
Unfortunately, almost all patients with advanced prostate cancer eventually progress to castration-resistant prostate cancer (CRPC)
after receiving endocrine therapy.
Moreover, mCRPC is the leading cause of patient mortality, with a 5-year survival rate of only about
15%.

Globally, prostate cancer is the second most common malignancy in men, after lung cancer
.
Prior to the marketing of Pluvicto, the preferred treatment regimen for patients who were not suitable for radical resection was androgen deprivation therapy, i.
e.
, castration levels
.
Unfortunately, almost all patients with advanced prostate cancer eventually progress to castration-resistant prostate cancer (CRPC)
after receiving endocrine therapy.
Moreover, mCRPC is the leading cause of patient mortality, with a 5-year survival rate of only about
15%.

Not only is Pluvuicto raising the bar for treatment benefit, but also Eli Lilly's Mounjaro, which represents the first new type of diabetes drug in nearly 10 years — integrating the effects of GIP and GLP-1 into a new molecule that reduces patients' weight
by 15 to 23 pounds compared to other diabetes drugs.

Not only is Pluvuicto raising the bar for treatment benefit, but also Eli Lilly's Mounjaro, which represents the first new type of diabetes drug in nearly 10 years — integrating the effects of GIP and GLP-1 into a new molecule that reduces patients' weight
by 15 to 23 pounds compared to other diabetes drugs.

Another breakthrough Eli Lilly brought to the industry was Oluminant
, the first alopecia areata treatment.
Although this is a common JAK inhibitor, it was not until the advent of Oluminant that the gap
in the treatment of alopecia areata was broken by this drug type.
The data showed that nearly 75% of adult patients with severe alopecia areata who responded to the treatment of Olumiant at 4 mg doses reached 90% of the scalp hair coverage at 52 weeks
.

Another breakthrough Eli Lilly brought to the industry was Oluminant
, the first alopecia areata treatment.
Although this is a common JAK inhibitor, it was not until the advent of Oluminant that the gap
in the treatment of alopecia areata was broken by this drug type.
The data showed that nearly 75% of adult patients with severe alopecia areata who responded to the treatment of Olumiant at 4 mg doses reached 90% of the scalp hair coverage at 52 weeks
.

Of course, in addition to MNC, Biotech's innovation should not be underestimated
.
In January, the bispecific fusion protein Kimmmtrak brought by Immunocore was approved for the treatment of HLA-A*02:01 positive adult metastatic uveal melanoma (mUM), the world's first TCR therapy
.

Of course, in addition to MNC, Biotech's innovation should not be underestimated
.
In January, the bispecific fusion protein Kimmmtrak brought by Immunocore was approved for the treatment of HLA-A*02:01 positive adult metastatic uveal melanoma (mUM), the world's first TCR therapy
.

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2022H2: Rare diseases such as ALS and CALD may have a turnaround

2022H2: RARE DISEASEs such as ALS, CALD or have a turnaround 2022H2: ALS, CALD and other rare diseases or have a turnaround 2022H2: ALS, CALD and other rare diseases or have a turnaround

Stepping on the tail of the first half of the year, Gilead resubmitted its listing application to the FDA for
long-acting HIV therapy Lenacapavir.
This is the first potentially approved HIV capsid inhibitor that only needs to be injected once a year and does not overlap with currently approved antiretroviral therapy (ART
).

Stepping on the tail of the first half of the year, Gilead resubmitted its listing application to the FDA for
long-acting HIV therapy Lenacapavir.
This is the first potentially approved HIV capsid inhibitor that only needs to be injected once a year and does not overlap with currently approved antiretroviral therapy (ART
).

However, the FDA has not yet accepted the NDA, and the approval time of Lenacapavir is unclear whether it can fall in the second half of the year
.
Leaving aside the uncertain Lenacapavir, over time, there are plenty of new drugs waiting for the FDA to give an answer in less than 6 months, especially for rare diseases
.

However, the FDA has not yet accepted the NDA, and the approval time of Lenacapavir is unclear whether it can fall in the second half of the year
.
Leaving aside the uncertain Lenacapavir, over time, there are plenty of new drugs waiting for the FDA to give an answer in less than 6 months, especially for rare diseases
.

Amyotrophic lateral sclerosis (ALS) was discussed
in the first half of the year.
In May, Radicava ORS (edaravone, oral suspension) developed by Mitsubishi Tanabe Pharmaceuticals' U.
S.
subsidiary was approved by
the FDA.
But that doesn't change the fact that
ALS is an incurable and rapidly progressing neurodegenerative disease.

Amyotrophic lateral sclerosis (ALS) was discussed
in the first half of the year.
In May, Radicava ORS (edaravone, oral suspension) developed by Mitsubishi Tanabe Pharmaceuticals' U.
S.
subsidiary was approved by
the FDA.
But that doesn't change the fact that
ALS is an incurable and rapidly progressing neurodegenerative disease.

The industry's hope is in the AMX0035 (Albrioza) from Amylyx
.
Albrioza, the first drug to show improved function and survival in ALS, was conditionally approved
in Canada in June.

The industry's hope is in the AMX0035 (Albrioza) from Amylyx
.
Albrioza, the first drug to show improved function and survival in ALS, was conditionally approved
in Canada in June.

The FDA has no shortage of opportunities, but it seems intent on slowing down
.
For the new drug with a special mechanism — inhibiting endoplasmic reticulum stress and mitochondrial dysfunction to protect motor neurons from damage — in March, the FDA expert committee considered its effectiveness questionable and voted 6:4 against recommending approval of AMX0035
.

The FDA has no shortage of opportunities, but it seems intent on slowing down
.
For the new drug with a special mechanism — inhibiting endoplasmic reticulum stress and mitochondrial dysfunction to protect motor neurons from damage — in March, the FDA expert committee considered its effectiveness questionable and voted 6:4 against recommending approval of AMX0035
.

After the PDUFA date was postponed by three months, AMX0035 will usher in a final approval decision
in September.

After the PDUFA date was postponed by three months, AMX0035 will usher in a final approval decision
in September.

Another "dead" rare disease is the BlueBird creature
.
The good news is that two gene therapies, Betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel( eli-cel, were approved by the FDA's expert committee last month
.

Another "dead" rare disease is the BlueBird creature
.
The good news is that two gene therapies, Betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel( eli-cel, were approved by the FDA's expert committee last month
.

Eli-cel targets early active cerebral adrenoleukodystrophy (CALD), which can rapidly lead to progressive, irreversible loss of nerve function and death
.
Currently, the only effective treatment for CALD is allogeneic hematopoietic stem cell transplantation, although it is estimated that more than 80% of relatives of CALD patients do not have a matching donor
.

Eli-cel targets early active cerebral adrenoleukodystrophy (CALD), which can rapidly lead to progressive, irreversible loss of nerve function and death
.
Currently, the only effective treatment for CALD is allogeneic hematopoietic stem cell transplantation, although it is estimated that more than 80% of relatives of CALD patients do not have a matching donor
.

As for beti-cel, it is intended to be used to treat patients with β thalassemia who require routine red blood cell transfusions
.
The therapy, which isolates hematopoietic stem cells from patients and uses adenoviral vectors to introduce modified β globulin genes that perform normal functions, is a potential one-time therapy gene therapy
.

As for beti-cel, it is intended to be used to treat patients with β thalassemia who require routine red blood cell transfusions
.
The therapy, which isolates hematopoietic stem cells from patients and uses adenoviral vectors to introduce modified β globulin genes that perform normal functions, is a potential one-time therapy gene therapy
.

Also of concern is Amiacus, who launched a two-drug combination (AT-GAA) to treat Pompe disease, which was also approved
in the second half of the year.
The miglustat in the portfolio has been approved for the treatment of Gaucher disease, and Amicus has also submitted NDA
for the treatment of Pompe disease.

Also of concern is Amiacus, who launched a two-drug combination (AT-GAA) to treat Pompe disease, which was also approved
in the second half of the year.
The miglustat in the portfolio has been approved for the treatment of Gaucher disease, and Amicus has also submitted NDA
for the treatment of Pompe disease.

With the FDA extension, the PDUFA dates for Miglustat and another drug in the portfolio, cilaglucosidase alfa, are set for August 29 and October 29, respectively
.

With the FDA extension, the PDUFA dates for Miglustat and another drug in the portfolio, cilaglucosidase alfa, are set for August 29 and October 29, respectively
.

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Between acceleration and caution, how does the FDA use balancing?

Between acceleration and caution, how does the FDA use balancing? Between acceleration and caution, how does the FDA use balancing? Between acceleration and caution, how does the FDA use balancing?

To drive innovative drugs to market, the FDA has established a series of accelerated approval procedures, including fast-track, breakthrough therapies, accelerated approvals, and priority reviews
.
In nature, fast-track, breakthrough therapies and priority reviews are drug-specific approvals, while accelerated approvals are drug review approval pathways
.

To drive innovative drugs to market, the FDA has established a series of accelerated approval procedures, including fast-track, breakthrough therapies, accelerated approvals, and priority reviews
.
In nature, fast-track, breakthrough therapies and priority reviews are drug-specific approvals, while accelerated approvals are drug review approval pathways
.

In terms of review, a total of 13 new drugs were approved by the FDA through priority review in the first half of this year, and 6 new drugs were awarded breakthrough therapies
.
This is in line
with the trend in recent years.
Combined with the new drugs approved by the FDA in 2017-2021, the average annual proportion of new drugs using accelerated procedures is more than 60%, especially in the past three years, and this proportion has increased
year by year.

In terms of review, a total of 13 new drugs were approved by the FDA through priority review in the first half of this year, and 6 new drugs were awarded breakthrough therapies
.
This is in line
with the trend in recent years.
Combined with the new drugs approved by the FDA in 2017-2021, the average annual proportion of new drugs using accelerated procedures is more than 60%, especially in the past three years, and this proportion has increased
year by year.

Behind the prosperity, there is no criticism
.
In June 2021, the FDA, unusually, accelerated the approval of Aduhelm, a new drug for the treatment of Alzheimer's disease by Bojian, despite the negative evaluation given by the expert committee, pushing the crisis of public trust in the FDA to a new high
.
In the space of a year, Aduhelm's commercial waterloo has further confirmed the inadequacy
of the initial accelerated approval.

Behind the prosperity, there is no criticism
.
In June 2021, the FDA, unusually, accelerated the approval of Aduhelm, a new drug for the treatment of Alzheimer's disease by Bojian, despite the negative evaluation given by the expert committee, pushing the crisis of public trust in the FDA to a new high
.
In the space of a year, Aduhelm's commercial waterloo has further confirmed the inadequacy
of the initial accelerated approval.

Previously, the British Medical Journal (BMJ) reported that up to 44% of the 253 drugs approved under the accelerated pathway have not been clinically proven to be effective
.
Medicare spent as much as $224 million on four anti-tumor drugs that were eventually withdrawn for accelerated approval in 2017-2019, according to
one study.

Previously, the British Medical Journal (BMJ) reported that up to 44% of the 253 drugs approved under the accelerated pathway have not been clinically proven to be effective
.
Medicare spent as much as $224 million on four anti-tumor drugs that were eventually withdrawn for accelerated approval in 2017-2019, according to
one study.

What the FDA has in front of us is how to rebuild credibility
.
In February, before becoming FDA director for the second time, Robert Califf promised that if the nomination was confirmed, he would take accelerated and "strong action" to require pharmaceutical companies to provide the scientific evidence
needed to expedite drug approval.

What the FDA has in front of us is how to rebuild credibility
.
In February, before becoming FDA director for the second time, Robert Califf promised that if the nomination was confirmed, he would take accelerated and "strong action" to require pharmaceutical companies to provide the scientific evidence
needed to expedite drug approval.

By April, the FDA held a meeting of the Expert Advisory Committee on Oncology Drugs (ODAC) to discuss and vote
on the design of future clinical trials of PI3K inhibitors in the field of blood cancer.
ODAC experts agree almost unanimously that approval of PI3K inhibitors needs to be based on randomized data rather than single-arm clinical trials
.
This may be seen as a response to
changes to the accelerated approval process.

By April, the FDA held a meeting of the Expert Advisory Committee on Oncology Drugs (ODAC) to discuss and vote
on the design of future clinical trials of PI3K inhibitors in the field of blood cancer.
ODAC experts agree almost unanimously that approval of PI3K inhibitors needs to be based on randomized data rather than single-arm clinical trials
.
This may be seen as a response to
changes to the accelerated approval process.

In May, the FDA issued new draft guidance detailing how to assess quality risks, sources of uncertainty, and possible mitigation strategies
.
According to the 12-page document, the FDA stepped up
its scrutiny of CMC's strategy.
In most cases, fda will not approve the application
if the product has unresolved quality issues.

In May, the FDA issued new draft guidance detailing how to assess quality risks, sources of uncertainty, and possible mitigation strategies
.
According to the 12-page document, the FDA stepped up
its scrutiny of CMC's strategy.
In most cases, fda will not approve the application
if the product has unresolved quality issues.

In addition to tightening the mouth, in the past six months, the FDA has also tried to provide guidance for more solid development of innovative therapies, such as the establishment of a rare neurodegenerative disease working group in the case of ALS, and the establishment of a public-private partnership
for rare neurodegenerative diseases.

In addition to tightening the mouth, in the past six months, the FDA has also tried to provide guidance for more solid development of innovative therapies, such as the establishment of a rare neurodegenerative disease working group in the case of ALS, and the establishment of a public-private partnership
for rare neurodegenerative diseases.

The ALS Science Strategy outlines a series of actions that the FDA will undertake over the next five years (ending June 2027) to address the obstacles and challenges
currently encountered in the development of ALS drugs.

The ALS Science Strategy outlines a series of actions that the FDA will undertake over the next five years (ending June 2027) to address the obstacles and challenges
currently encountered in the development of ALS drugs.

In the short term, FDA is focused on building the infrastructure
needed to implement ALS's science strategy.
According to the plan, the Rare Neurodegenerative Diseases Working Group will spin out a dedicated ALS working group to advance medical development in the field; In the medium term, the FDA will focus on patient-centered drug development, encourage data sharing, and develop research data standards; In the long term, the FDA will strengthen the design and implementation of clinical trials and explore innovative trial designs
.

In the short term, FDA is focused on building the infrastructure
needed to implement ALS's science strategy.
According to the plan, the Rare Neurodegenerative Diseases Working Group will spin out a dedicated ALS working group to advance medical development in the field; In the medium term, the FDA will focus on patient-centered drug development, encourage data sharing, and develop research data standards; In the long term, the FDA will strengthen the design and implementation of clinical trials and explore innovative trial designs
.

Obviously, the above-mentioned distant water cannot save the near fire
.
But perhaps for the FDA at this time, slow is fast, less is more
.

Obviously, the above-mentioned distant water cannot save the near fire
.
But perhaps for the FDA at this time, slow is fast, less is more
.